UNITED STATES

SECURITIES AND EXCHANGE COMMISSION

Washington D.C. 20549

FORM 10-Q

(Mark One)

☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended ~~March~~June 3130, 2026 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from _______ to _______ Commission file number: 001-36294

uniQure N.V.

(Exact name of Registrant as specified in its charter)

The Netherlands

(State or other jurisdiction of incorporation or organization)

Not applicable

(I.R.S. Employer Identification No.)

Paasheuvelweg ~~25a~~25

1105 BP Amsterdam, The Netherlands

(Address of principal executive offices) (Zip Code)

+31-20-240-6000

(Registrant’s telephone number, including area code)

Securities registered pursuant to Section 12(b) of the Act:


Title of each class:	Trading Symbol(s)	Name of each exchange on which registered
Ordinary Shares, par value €0.05	QURE	The Nasdaq Stock Market LLC (The Nasdaq Global Select Market)

Indicate by check mark whether the registrant (1) has filed all reports required to be filed by Section 13 or 15(d) of the Securities Exchange Act of 1934 during the preceding 12 months (or for such shorter period that the registrant was required to file such reports), and (2) has been subject to such filing requirements for the past 90 days. Yes ⌧ No ◻.

Indicate by check mark whether the registrant has submitted electronically every Interactive Data File required to be submitted pursuant to Rule 405 of Regulation S-T (§ 232.405 of this chapter) during the preceding 12 months (or for such shorter period that the registrant was required to submit such files). Yes ⌧ No ◻.

Indicate by check mark whether the registrant is a large accelerated filer, an accelerated filer, a non-accelerated filer, smaller reporting company or an emerging growth company. See the definitions of “large accelerated filer” “accelerated filer” and “smaller reporting company” and “emerging growth company” in Rule 12b-2 of the Exchange Act.

Large accelerated filer ⌧

Accelerated filer ◻

Non-accelerated filer ◻

Smaller reporting company ☐

Emerging growth company ☐

If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13 (a) of the Exchange Act ◻

Indicate by check mark whether the registrant is a shell company (as defined in Rule 12b-2 of the Exchange Act.) Yes ☐ No ⌧

As of ~~May~~July 124, 2026, the registrant had 6369,~~067~~366,~~485~~412 ordinary shares, par value €0.05, outstanding.

Table of Contents

TABLE OF CONTENTS

		Page
	PART I – FINANCIAL INFORMATION

Item 1	Financial Statements	2

Item 2	Management’s Discussion and Analysis of Financial Condition and Results of Operations	1820

Item 3	Quantitative and Qualitative Disclosures About Market Risk	3136

Item 4	Controls and Procedures	3137

	PART II – OTHER INFORMATION

Item 1	Legal Proceedings	3237

Item 1A	Risk Factors	3238

Item 2	Unregistered Sales of Equity Securities, Use of Proceeds, and Issuer Purchases of Equity Securities	3340

Item 3	Defaults Upon Senior Securities	3340

Item 4	Mine Safety Disclosures	3340

Item 5	Other Information	3340

Item 6	Exhibits	3341

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SPECIAL CAUTIONARY NOTICE REGARDING FORWARD-LOOKING STATEMENTS

This Quarterly Report on Form 10-Q contains “forward-looking statements” within the meaning of the federal securities laws. Forward-looking statements are based on our current expectations of future events and many of these statements can be identified using terminology such as “believes,” “expects,” “anticipates,” “plans,” “may,” “will,” “projects,” “continues,” “estimates,” “potential,” “opportunity” and similar expressions. These forward-looking statements involve known and unknown risks, uncertainties and other factors that may cause our actual results, levels of activity, performance or achievements to be materially different from the information expressed or implied by these forward-looking statements. These forward-looking statements include, without limitation, statements concerning: our financial position and performance, revenues, costs, expenses, liquidity, uses of cash and capital requirements; our need for additional financing or the time period for which our existing cash resources will be sufficient to meet our operating requirements; the success, progress, number, scope, cost, duration, timing or results of our research and development activities, preclinical and clinical trials, including the timing for initiation, completion or availability of results from any preclinical studies and clinical trials or for the submission, review or approval of any regulatory filing, including ~~the~~with ~~timing~~respect ofto the Company’s Biologics License Application for AMT-130 to the United States Food and Drug Administration (the “FDA”) and Marketing Authorization Application for AMT-130 to the United Kingdom’s Medicines and Healthcare products Regulatory Agency (“MHRA”); the anticipated or ultimate outcome, and timing, of interactions with regulatory agencies, including the FDA and MHRA; our plans to conduct a confirmatory study for AMT-130; the timing of, and our ability to, obtain and maintain regulatory approvals for any of our product candidates; the potential benefits ~~that~~and ~~may~~clinical, betherapeutic ~~derived~~and ~~from~~commercial ~~any~~potential of our product candidates; our strategies, prospects, plans, goals, expectations, forecasts or objectives; the success of our collaborations with third parties; our ability to identify and develop new product candidates and technologies; our intellectual property position; our commercialization, marketing and manufacturing capabilities and strategy, including plans for potential commercialization of AMT-130; our ability to achieve long-term growth; and developments and projections relating to our competitors in the industry.

Forward-looking statements are only predictions based on management’s current views and assumptions and involve risks and uncertainties, and actual results could differ materially from those projected or implied. The most significant factors known to us that could materially adversely affect our business, operations, industry, financial position or future financial performance include those discussed in Part II, Item 1A “Risk Factors,” as well as those discussed in Part I, Item 2 “Management’s Discussion and Analysis of Financial Condition and Results of Operations” and elsewhere in this Quarterly Report on Form 10-Q, as well as other factors which may be identified from time to time in our other filings with the Securities and Exchange Commission (the “SEC”), including our most recent Annual Report on Form 10-K filed with the SEC on March 2, 2026 (the “Annual Report”), our Quarterly Reports on Form 10-Q, Current Reports on Form 8-K, or in the documents where such forward-looking statements appear. You should carefully consider that information before you make an investment decision.

You should not place undue reliance on these forward-looking statements, which speak only as of the date that they were made. Our actual results or experience could differ significantly from those anticipated in the forward-looking statements and from historical results, due to the risks and uncertainties described in this Quarterly Report on Form 10-Q and in our Annual Report, including in “Part I, Item 1A. Risk Factors,” as well as others that we may consider immaterial or do not anticipate at this time. These cautionary statements should be considered in connection with any written or oral forward-looking statements that we may make in the future or may file or furnish with the SEC. We do not undertake any obligation to release publicly any revisions to these forward-looking statements after completion of the filing of this Quarterly Report on Form 10-Q to reflect later events or circumstances or to reflect the occurrence of unanticipated events. All forward-looking statements attributable to us are expressly qualified in their entirety by these cautionary statements.

In addition, with respect to all our forward-looking statements, we claim the protection of the safe harbor for forward-looking statements contained in the Private Securities Litigation Reform Act of 1995.

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Part I – FINANCIAL INFORMATION

Item 1.Financial Statements

uniQure N.V.

UNAUDITED CONSOLIDATED BALANCE SHEETS


	~~March~~June 3130,		December 31,
	2026		2025
	(in thousands, except share and per share amounts)
Current assets
Cash and cash equivalents	$	~~139~~413,~~994~~027	$	80,240
Current investment securities		~~446~~397,~~556~~308		542,301
Accounts receivable		35,~~562~~778		5,863
Prepaid expenses		1610,~~589~~245		20,506
Other current assets and receivables		97,~~043~~911		7,076
Total current assets		~~615~~834,~~744~~269		655,986
Non-current assets
Property, plant and equipment, net		1211,~~014~~376		13,800
Other investments		30,~~150~~267		30,237
Operating lease right-of-use assets		1211,~~261~~606		12,525
Intangible assets, net		6962,~~990~~101		72,790
Goodwill		24,~~811~~587		25,355
Deferred tax assets, net		86,~~194~~362		8,654
Other non-current assets		54,~~542~~535		5,561
Total non-current assets		~~162~~150,~~962~~834		168,922
Total assets	$	~~778~~985,~~706~~103	$	824,908
Current liabilities
Accounts payable	$	4,~~399~~979	$	5,170
Accrued expenses and other current liabilities		4351,~~201~~133		41,292
Liability related to pre-funded warrants		824,~~605~~242		12,595
Current portion of operating lease liabilities		2,~~992~~991		3,862
Total current liabilities		5983,~~197~~345		62,919
Non-current liabilities
Long-term debt		4950,~~942~~147		49,699
Liability from royalty financing agreement		~~482~~489,~~334~~333		473,199
Operating lease liabilities, net of current portion		109,~~388~~696		9,832
Contingent consideration		1718,~~029~~113		18,736
Deferred tax liability, net		7,~~796~~726		7,967
Other non-current liabilities, net of current portion		2,~~677~~693		3,655
Total non-current liabilities		~~570~~577,~~166~~708		563,088
Total liabilities		~~629~~661,~~363~~053		626,007
Commitments and contingencies
Shareholders' equity
Ordinary shares, €0.05 par value: 80,000,000 shares authorized at March 31, 2026, and December 31, 2025, and 63,033,249 and 62,336,717 shares issued and outstanding at March 31, 2026, and December 31, 2025, respectively.		3,728		3,688
Ordinary shares, €0.05 par value: 100,000,000 shares authorized at June 30, 2026, and 80,000,000 at December 31, 2025, and 69,334,839 and 62,336,717 shares issued and outstanding at June 30, 2026, and December 31, 2025, respectively.		4,086		3,688
Additional paid-in-capital		1,587,971		1,582,371
Additional paid-in capital		1,841,745		1,582,371
Accumulated other comprehensive loss		(5958,~~885~~251)		(58,222)
Accumulated deficit		(1,~~382~~463,~~471~~530)		(1,328,936)
Total shareholders' equity		~~149~~324,~~343~~050		198,901
Total liabilities and shareholders' equity	$	~~778~~985,~~706~~103	$	824,908

The accompanying notes are an integral part of these unaudited consolidated financial statements.

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uniQure N.V.

UNAUDITED CONSOLIDATED STATEMENTS OF OPERATIONS AND
COMPREHENSIVE LOSS


	Three months ended ~~March~~June 3130,				Six months ended June 30,
	2026		2025		2026		2025
	(in thousands, except share and per share amounts)				(in thousands, except share and per share amounts)
License revenues	$	35,~~562~~841	$	15,~~567~~262	$	9,403	$	6,829
Total revenues		35,~~562~~841		15,~~567~~262		9,403		6,829
Operating expenses:
Cost of license revenues		(~~219~~350)		(~~197~~656)		(569)		(853)
Research and development expenses		(2933,~~176~~964)		(3635,~~140~~383)		(63,140)		(71,523)
Selling, general and administrative expenses		(2017,~~068~~367)		(1013,~~908~~500)		(37,435)		(24,408)
Total operating expenses		(4951,~~463~~681)		(4749,~~245~~539)		(101,144)		(96,784)
Other income		1,~~632~~598		82,~~306~~597		3,230		10,903
Other expense		(17,~~451~~961)		(12,~~959~~185)		(9,412)		(4,144)
Loss from operations		(4552,~~720~~203)		(3943,~~331~~865)		(97,923)		(83,196)
Interest income		5,~~229~~051		43,~~127~~524		10,280		7,651
Interest expense		(14,~~031~~366)		(15,~~109~~591)		(28,397)		(30,700)
Foreign currency (losses) / gains, net		(21,~~294~~730)		718,~~172~~638		(4,024)		25,810
Other non-operating gains, net		3,769		—
Other non-operating (losses), net		(15,970)		—		(12,201)		—
Loss before income tax expense	$	(5379,~~047~~218)	$	(4337,~~141~~294)	$	(132,265)	$	(80,435)
Income tax expense		(~~488~~1,841)		(~~496~~425)		(2,329)		(921)
Net loss	$	(5381,~~535~~059)	$	(4337,~~637~~719)	$	(134,594)	$	(81,356)
Other comprehensive loss:
Foreign currency translation losses, net		(1,663)		(1,005)
Foreign currency translation gains / (losses), net		1,634		(5,098)		(29)		(6,103)
Total comprehensive loss	$	(5579,~~198~~425)	$	(4442,~~642~~817)	$	(134,623)	$	(87,459)
Loss per ordinary share - basic and diluted
Basic and diluted net loss per ordinary share	$	(01.8522)	$	(0.8269)	$	(2.09)	$	(1.49)
Weighted average shares - basic and diluted		6266,~~742~~243,~~847~~879		5354,~~110~~807,~~580~~967		64,503,035		54,540,423

The accompanying notes are an integral part of these unaudited consolidated financial statements.

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uniQure N.V.

UNAUDITED CONSOLIDATED STATEMENTS OF SHAREHOLDERS’ ~~EQUITYFOR~~EQUITY / (DEFICIT)FOR THE THREE MONTHS ENDED ~~MARCH~~JUNE 3130, 2026 AND 2025


						Accumulated
				Additional		other				Total
	Ordinary shares			paid-in		comprehensive		Accumulated		shareholders’
	No. of shares	Amount		capital		loss		deficit		equity
	(in thousands, except share and per share amounts)
Balance at December 31, 2024	48,988,087	$	2,945	$	1,173,068	$	(52,800)	$	(1,129,965)	$	(6,752)
Loss for the period	—		—		—		—		(43,637)		(43,637)
Other comprehensive loss, net	—		—		—		(1,005)		—		(1,005)
Follow-on public offering	5,073,529		261		80,250		—		—		80,511
Exercises of share options	15,928		1		158		—		—		159
Restricted and performance share units distributed during the period	620,935		33		(33)		—		—		—
Share-based compensation expense	—		—		4,410		—		—		4,410
Balance at March 31, 2025	54,698,479	$	3,240	$	1,257,853	$	(53,805)	$	(1,173,602)	$	33,686

Balance at December 31, 2025	62,336,717	$	3,688	$	1,582,371	$	(58,222)	$	(1,328,936)	$	198,901
Loss for the period	—		—		—		—		(53,535)		(53,535)
Other comprehensive loss, net	—		—		—		(1,663)		—		(1,663)
Exercises of share options	25,308		1		384		—		—		385
Restricted share units distributed during the period	657,285		38		(38)		—		—		—
Issuance of ordinary shares relating to employee stock purchase plan	13,939		1		164		—		—		165
Share-based compensation expense	—		—		5,090		—		—		5,090
Balance at March 31, 2026	63,033,249	$	3,728	$	1,587,971	$	(59,885)	$	(1,382,471)	$	149,343

The accompanying notes are an integral part of these unaudited consolidated financial statements.


										Accumulated
							Additional			other						Total
		Ordinary shares					paid-in			comprehensive			Accumulated			shareholders’
		No. of shares		Amount			capital			gain / (loss)			deficit			equity / (deficit)

		(in thousands, except share and per share amounts)
Balance at March 31, 2025		54,698,479		$	3,240		$	1,257,853		$	(53,805)		$	(1,173,602)		$	33,686
Loss for the period		—			—			—			—			(37,719)			(37,719)
Other comprehensive loss, net		—			—			—			(5,098)			—			(5,098)
Exercise of share options		5,312			—			44			—			—			44
Restricted share units distributed during the period		161,882			10			(10)			—			—			—
Share-based compensation expense		—			—			5,084			—			—			5,084
Balance at June 30, 2025		54,865,673		$	3,250		$	1,262,971		$	(58,903)		$	(1,211,321)		$	(4,003)

Balance at March 31, 2026		63,033,249		$	3,728		$	1,587,971		$	(59,885)		$	(1,382,471)		$	149,343
Loss for the period		—			—			—			—			(81,059)			(81,059)
Other comprehensive gain, net		—			—			—			1,634			—			1,634
Follow-on public offering		5,686,813			323			242,359			—			—			242,681
Exercises of share options		385,541			23			6,412			—			—			6,435
Restricted share units distributed during the period		229,236			13			(13)			—			—			—
Share-based compensation expense		—			—			5,016			—			—			5,016
Balance at June 30, 2026		69,334,839		$	4,086		$	1,841,745		$	(58,251)		$	(1,463,530)		$	324,050

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uniQure N.V.

UNAUDITED CONSOLIDATED STATEMENTS OF ~~CASH~~SHAREHOLDERS’ ~~FLOWS~~EQUITY / (DEFICIT)FOR THE SIX MONTHS ENDED JUNE 30, 2026 AND 2025


	Three months ended March 31,
	2026		2025
	(in thousands)
Cash flows from operating activities
Net loss	$	(53,535)	$	(43,637)
Adjustments to reconcile net loss to net cash used in operating activities:
Depreciation and amortization		2,956		4,527
Amortization of discount on investment securities		(4,335)		(2,117)
Share-based compensation expense		5,090		4,410
Royalty financing agreement interest expense, net of interest paid		7,173		8,358
Deferred tax expense		458		496
Changes in fair value of contingent consideration		(1,338)		1,216
Changes in fair value of liability related to pre-funded warrants		(3,769)		—
Unrealized foreign exchange losses / (gains), net		2,178		(7,105)
Other items, net		(242)		(3,971)
Changes in operating assets and liabilities:
Accounts receivable, prepaid expenses, and other current assets and receivables		3,780		(2,979)
Accounts payable		(557)		(2,093)
Accrued expenses, other liabilities, and operating leases		3,898		(1,205)
Net cash used in operating activities		(38,243)		(44,100)
Cash flows from investing activities
Proceeds on maturity of debt securities		98,700		85,000
Investment in debt securities		—		(64,775)
Purchases of property, plant, and equipment		(140)		(126)
Net cash generated from investing activities		98,560		20,099
Cash flows from financing activities
Proceeds from follow-on public offering of ordinary shares, net of issuance costs		—		80,511
Proceeds from issuance of ordinary shares related to employee stock options and purchase plans		550		159
Net cash generated from financing activities		550		80,670
Currency effect on cash, cash equivalents and restricted cash		(1,132)		1,660
Net increase in cash, cash equivalents and restricted cash		59,735		58,329
Cash, cash equivalents and restricted cash at the beginning of period		81,801		160,329
Cash, cash equivalents and restricted cash at the end of period	$	141,536	$	218,658
Cash and cash equivalents	$	139,994	$	217,229
Restricted cash related to leasehold and other deposits		1,542		1,429
Total cash, cash equivalents and restricted cash	$	141,536	$	218,658
Supplemental cash flow disclosures:
Cash paid for interest	$	(6,720)	$	(6,491)


										Accumulated
							Additional			other						Total
		Ordinary shares					paid-in			comprehensive			Accumulated			shareholders’
		No. of shares		Amount			capital			(loss)			deficit			equity / (deficit)

		(in thousands, except share and per share amounts)
Balance at December 31, 2024		48,988,087		$	2,945		$	1,173,068		$	(52,800)		$	(1,129,965)		$	(6,752)
Loss for the period		—			—			—			—			(81,356)			(81,356)
Other comprehensive loss, net		—			—			—			(6,103)			—			(6,103)
Follow-on public offering		5,073,529			261			80,250			—			—			80,511
Exercises of share options		21,240			1			202			—			—			203
Restricted and performance share units distributed during the period		782,817			43			(43)			—			—			—
Share-based compensation expense		—			—			9,494			—			—			9,494
Balance at June 30, 2025		54,865,673		$	3,250		$	1,262,971		$	(58,903)		$	(1,211,321)		$	(4,003)

Balance at December 31, 2025		62,336,717		$	3,688		$	1,582,371		$	(58,222)		$	(1,328,936)		$	198,901
Loss for the period		—			—			—			—			(134,594)			(134,594)
Other comprehensive loss, net		—			—			—			(29)			—			(29)
Follow-on public offering		5,686,813			323			242,359			—			—			242,681
Exercises of share options		410,849			24			6,796			—			—			6,820
Restricted share units distributed during the period		886,521			51			(51)			—			—			—
Issuance of ordinary shares relating to employee stock purchase plan		13,939			1			164			—			—			165
Share-based compensation expense		—			—			10,106			—			—			10,106
Balance at June 30, 2026		69,334,839		$	4,086		$	1,841,745		$	(58,251)		$	(1,463,530)		$	324,050

The accompanying notes are an integral part of these unaudited consolidated financial statements.

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uniQure N.V.

UNAUDITED CONSOLIDATED STATEMENTS OF CASH FLOWS


		Six months ended June 30,
		2026			2025

		(in thousands)
Cash flows from operating activities
Net loss		$	(134,594)		$	(81,356)
Adjustments to reconcile net loss to net cash used in operating activities:
Depreciation and amortization			7,863			8,157
Amortization of discount on investment securities			(7,907)			(4,163)
Share-based compensation expense			10,106			9,494
Royalty financing agreement interest expense, net of interest paid			17,384			20,626
Deferred tax expense			2,329			921
Changes in fair value of contingent consideration			(82)			3,470
Changes in fair value of liability related to pre-funded warrants			12,201			—
Unrealized foreign exchange losses / (gains), net			8,329			(25,163)
Other items, net			(2,426)			(3,651)
Changes in operating assets and liabilities:
Accounts receivable, prepaid expenses, and other current assets and receivables			8,846			(6,761)
Accounts payable			68			(2,297)
Accrued expenses, other liabilities, and operating leases			11,064			(3,272)
Net cash used in operating activities			(66,819)			(83,995)
Cash flows from investing activities
Proceeds on maturity of debt securities			384,981			213,763
Investments in debt securities			(232,550)			(120,205)
Purchases of property, plant, and equipment			(612)			(386)
Net cash generated from investing activities			151,819			93,172
Cash flows from financing activities
Proceeds from follow-on public offering of ordinary shares, net of issuance costs			242,681			80,511
Proceeds from issuance of ordinary shares related to employee stock options and purchase plans			6,985			203
Net cash generated from financing activities			249,666			80,714
Currency effect on cash, cash equivalents and restricted cash			(1,906)			5,052
Net increase in cash, cash equivalents and restricted cash			332,760			94,943
Cash, cash equivalents and restricted cash at the beginning of period			81,801			160,329
Cash, cash equivalents and restricted cash at the end of period		$	414,561		$	255,272
Cash and cash equivalents		$	413,027		$	253,778
Restricted cash related to leasehold and other deposits			1,534			1,494
Total cash, cash equivalents and restricted cash		$	414,561		$	255,272
Supplemental cash flow disclosures:
Cash paid for interest		$	(11,494)		$	(9,652)

The accompanying notes are an integral part of these unaudited consolidated financial statements.

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uniQure N.V.

NOTES TO CONSOLIDATED FINANCIAL STATEMENTS

(Unaudited)

1General business information

uniQure N.V. (the “Company”) was incorporated on January 9, 2012, as a private company with limited liability (besloten vennootschap met beperkte aansprakelijkheid) under the laws of the Netherlands. The Company is a leader in the field of gene therapy and seeks to deliver to patients suffering from rare and other devastating diseases single treatments with potentially curative results. The Company’s business was founded in 1998 and was initially operated through its predecessor company, Amsterdam Molecular Therapeutics Holding N.V. (“AMT”). In 2012, AMT undertook a corporate reorganization, pursuant to which uniQure B.V. acquired the entire business and assets of AMT and completed a share-for-share exchange with the shareholders of AMT. Effective February 10, 2014, in connection with its initial public offering, the Company converted into a public company with limited liability (naamloze vennootschap) and changed its legal name from uniQure B.V. to uniQure N.V.

The Company is registered in the trade register of the Dutch Chamber of Commerce (Kamer van Koophandel) in Amsterdam, the Netherlands under number 54385229. The Company’s headquarters are in Amsterdam, the Netherlands, its registered office is located at Paasheuvelweg ~~25a~~25, Amsterdam 1105 BP, the Netherlands and its telephone number is +31 20 240 6000.

The Company’s ordinary shares are listed on the Nasdaq Global Select Market and trade under the symbol “QURE”.

2Summary of significant accounting policies

2.1Basis of preparation

The Company prepared these unaudited consolidated financial statements in compliance with generally accepted accounting principles in the United States (“U.S. GAAP”) and applicable rules and regulations of the United States Securities and Exchange Commission (the “SEC”) regarding interim financial reporting. Any reference in these notes to applicable guidance is meant to refer to authoritative U.S. GAAP as found in the Accounting Standards Codification (“ASC”) and Accounting Standards Updates of the Financial Accounting Standards Board.

The unaudited consolidated financial statements are presented in United States (“U.S.”) dollars, except where otherwise indicated. Transactions denominated in currencies other than U.S. dollars are presented in the transaction currency with the U.S. dollar amount included in ~~parenthesis~~parentheses, converted at the foreign exchange rate as of the transaction date.

2.2Unaudited interim financial information

The interim financial statements and related disclosures are unaudited, have been prepared on the same basis as the annual financial statements and, in the opinion of management, reflect all adjustments, which include only normal recurring adjustments, necessary for a fair statement of the financial position, results of operations and changes in financial position for the period presented.

Certain information and footnote disclosures normally included in annual financial statements prepared in accordance with U.S. GAAP have been omitted. The results of operations for the three and six months ended ~~March~~June 3130, 2026, are not necessarily indicative of the results to be expected for the full year ending December 31, 2026, or for any other future year or interim period. The accompanying financial statements should be read in conjunction with the audited financial statements and the related notes thereto included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2025 filed by the Company with the SEC on March 2, 2026 (the “Annual Report”).

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2.3Use of estimates

The preparation of the financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities,; the disclosure of contingent liabilities at the date of the financial statements; and the reported amounts of revenues, expenses, and tax -related items during the reporting period. Actual results could differ from those estimates.

2.4Accounting policies

The principal accounting policies applied in the preparation of these unaudited consolidated financial statements are described in the Company’s audited financial statements as of and for the year ended December 31, 2025, and the notes thereto, which are included in the Annual Report. There have been no material changes in the Company’s significant accounting policies during the ~~three~~six months ended ~~March~~June 3130, 2026.

2.5Recent accounting pronouncements

There have been no new accounting pronouncements or changes to accounting pronouncements during the ~~three~~six months ended ~~March~~June 3130, 2026, as compared to the recent accounting pronouncements described in Note 2.3.29 of the Annual Report, which could be expected to materially impact the Company’s unaudited consolidated financial statements.

3	Current investment securities

The following tables summarize the Company’s investments in government debt securities as of ~~March~~June 3130, 2026 and December 31, 2025:


	AtJune ~~March 31~~30, 2026
	Amortized cost		Gross unrealized holding gains		Gross unrealized holding losses		Estimated fair value
	(in thousands)
Current investments securities:
Government debt securities (held-to-maturity)	$	~~446~~397,~~556~~308	$	~~295~~30	$	—	$	~~446~~397,~~851~~338
Total	$	~~446~~397,~~556~~308	$	~~295~~30	$	—	$	~~446~~397,~~851~~338

	At December 31, 2025
	Amortized cost		Gross unrealized holding gains		Gross unrealized holding losses		Estimated fair value
	(in thousands)
Current investments securities:
Government debt securities (held-to-maturity)	$	542,301	$	308	$	—	$	542,609
Total	$	542,301	$	308	$	—	$	542,609

The Company invests in short-term U.S. and European government debt securities with high investment credit ratings. The U.S. and European government bonds are U.S. dollar and euro (€, or EUR) denominated, respectively. Investment securities with original maturities of 90 days or less when purchased are presented within cash and cash equivalents and measured at amortized cost. Inputs to the fair value of the investments are considered Level 2 inputs.

As of ~~March~~June 3130, 2026 and December 31, 2025, no expected credit loss was identified with respect to the investment securities.

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4	Fair value measurement

The Company measures certain financial assets and liabilities at fair value, either upon initial recognition or for subsequent accounting or reporting. ASC 820, Fair Value Measurement requires disclosure of the methodologies used in determining the reported fair values and establishes a hierarchy of inputs used when available. The three levels of the fair value hierarchy are described below:

Level 1 – Valuations based on unadjusted quoted prices in active markets for identical assets or liabilities that the Company can access at the measurement date.

Level 2 – Valuations based on quoted prices for similar assets or liabilities in markets that are not active or models for which the inputs are observable, either directly or indirectly.

Level 3 – Valuations that require inputs that reflect the Company’s own assumptions that are both significant to the fair value measurement and are unobservable.

To the extent that valuation is based on models or inputs that are less observable or unobservable in the market, the determination of fair value requires more judgment. Accordingly, the degree of judgment exercised by the Company in determining fair value is greatest for instruments categorized as Level 3. A financial instrument’s level within the fair value hierarchy is based on the lowest level of any input that is significant to the fair value measurement.

The carrying amount of cash and cash equivalents, accounts receivable from licensing and collaboration partners, other assets, accounts payable, accrued expenses and other current liabilities reflected in the Unaudited Consolidated Balance Sheets approximate their fair values due to their short-term maturities.

The Company’s material financial assets include cash and cash equivalents, restricted cash and investment securities. Cash and cash equivalents and restricted cash are measured at fair value using Level 1 inputs. Restricted cash is included in Other non-current assets within the ~~unaudited~~Unaudited Consolidated Balance Sheets. Investment securities are measured at amortized cost.

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The following table sets forth the Company’s assets and liabilities that are required to be measured at fair value on a recurring basis as of ~~March~~June 3130, 2026 and December 31, 2025:


	Quoted pricesin activemarkets(Level 1)		Significant otherobservableinputs(Level 2)		Significantunobservableinputs(Level 3)		Total		Classification in Unaudited ConsolidatedBalance Sheets
		(in thousands)
Balance at December 31, 2025
Assets:
Cash and cash equivalents	$	80,240	$	—	$	—	$	80,240	Cash and cash equivalents
Restricted cash		1,561		—		—		1,561	Other non-current assets
Total assets	$	81,801	$	—	$	—	$	81,801
Liabilities:
Liability related to pre-funded warrants		—		12,595		—		12,595	Liability related to pre-funded warrants
Contingent consideration		—		—		18,736		18,736	Contingent consideration
Consideration for post-acquisition services		—		—		638		638	Other non-current liabilities
Total liabilities	$	—	$	12,595	$	19,374	$	31,969
Balance at March 31, 2026
Balance at June 30, 2026
Assets:
Cash and cash equivalents	$	~~139~~413,~~994~~027	$	—	$	—	$	~~139~~413,~~994~~027	Cash and cash equivalents
Restricted cash		1,~~542~~534		—		—		1,~~542~~534	Other non-current assets
Total assets	$	~~141~~414,~~536~~561	$	—	$	—	$	~~141~~414,~~536~~561
Liabilities:
Liability related to pre-funded warrants		—		824,~~605~~242		—		824,~~605~~242	Liability related to pre-funded warrants
Contingent consideration		—		—		1718,~~029~~113		1718,~~029~~113	Contingent consideration
Consideration for post-acquisition services		—		—		~~580~~617		~~580~~617	Other non-current liabilities
Total liabilities	$	—	$	824,~~605~~242	$	1718,~~609~~730	$	2642,~~214~~972

Contingent consideration

The Company is required to pay up to EUR 143.1 million (or $~~164~~163.50 million based on the foreign exchange rate on ~~March~~June 3130, 2026) to the former shareholders of uniQure France SAS (formerly Corlieve Therapeutics SAS) upon the achievement of the remaining contractually defined milestones related to the development of AMT-260 in connection with the Company’s acquisition of uniQure France SAS.

The fair value of the contingent consideration as of ~~March~~June 3130, 2026 was EUR 1415.89 million ($1718.01 million) (December 31, 2025: EUR 15.9 million ($18.7 million)) using discount rates of approximately 1110.51% to 11.8% (December 31, 2025: 11.8%).

If, as of ~~March~~June 3130, 2026, the Company had assumed a 100% likelihood of AMT-260 advancing into a Phase III clinical study, then the fair value of the contingent consideration would have increased to EUR 4751.83 million ($5558.04 million). If, as of ~~March~~June 3130, 2026, the Company had assumed that it would discontinue development of the AMT-260 program, then the contingent consideration would have been released to income.

~~The following table presents the changes in fair value of the contingent consideration (presented within non-current liabilities) between December 31, 2025 and March 31, 2026:~~10

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The following table presents the changes in fair value of the contingent consideration (presented within non-current liabilities) between December 31, 2025 and June 30, 2026:


	Amount of
	contingent
	consideration
	(in thousands)
Balance at December 31, 2025	$	18,736
Unrealized change in fair value (presented within Research and development expenses)		(~~1,338~~82)
Currency translation effects		(~~369~~541)
Balance at March 31, 2026	$	17,029
Balance at June 30, 2026	$	18,113

9The Company classified the total contingent consideration liability as non-current as of June 30, 2026 and December 31, 2025. The classification of the contingent consideration within the Company’s Unaudited Consolidated Balance Sheets between current and non-current liabilities is based upon the Company’s best estimate of the timing of settlement of the remaining relevant milestones.

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The Company classified the total contingent consideration liability as non-current as of March 31, 2026 and December 31, 2025. The classification of the contingent consideration within the Company’s Unaudited Consolidated Balance Sheets between current and non-current liabilities is based upon the Company’s best estimate of the timing of settlement of the remaining relevant milestones.

Liability related to pre-funded warrants

In September 2025, the Company completed a follow-on public offering which included the issuance of pre-funded warrants to purchase 526,316 of the Company’s ordinary shares at the public offering price of $47.50 per ordinary share, less a $0.0001 per ordinary share exercise price for each pre-funded warrant (“Pre-Funded Warrant(s)”). The obligation to issue ordinary shares upon the exercise of the Pre-Funded Warrants is classified as a liability related to pre-funded warrants.

The Pre-Funded Warrants are not exchange-quoted and are measured using a valuation technique whose significant inputs are observable; therefore, the fair value measurement is classified within Level 2 of the fair value hierarchy.


	Liability related
	to pre-funded
	warrants
	(in thousands)
Balance at December 31, 2025	$	12,595
Unrealized change in fair value (presented within Other non-operating gains, net)		(3,769)
Unrealized increase in fair value (presented within Other non-operating (losses), net)		12,201
Currency translation effects		(~~221~~554)
Balance at March 31, 2026	$	8,605
Balance at June 30, 2026	$	24,242

5	Accrued expenses and other current liabilities

Accrued expenses and other current liabilities include the following items:


	~~March~~June 3130,		December 31,
	2026		2025
	(in thousands)
Accruals for goods received from and services provided by vendors-not yet billed	$	23,493		20,490
Accruals for goods received from and services provided by vendors, not yet billed	$	24,684	$	20,490
Personnel-related accruals and liabilities		10,126	$	9,941
Personnel-related accruals and liabilities (As of June 30, 2026, $7.2 million (December 31, 2025: $0.0 million) related to cash proceeds from employees' stock option exercises)		16,654		9,941
Current portion of firm purchase commitment liability (refer to Note 13, "Commitments and contingencies")		5,796		5,102
Liability owed to the Purchaser pursuant to the Royalty Financing Agreement (refer to Note 7, "Royalty Financing Agreement")		5,778		5,523
Liability owed to the Purchaser pursuant to the Royalty Financing Agreement (refer to Note 7, "Royalty Financing Agreement")		3,562		5,523
Current portion of firm purchase commitment liability (including Genezen CSA termination payment)		3,808		5,102
Other current liabilities		~~224~~209		236
Total	$	4351,~~201~~133	$	41,292

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In April 2026, the Company entered into an agreement to terminate the commercial supply agreement (the “Genezen CSA”) with Genezen MA, Inc. (together with Genezen Holdings Inc., “Genezen”). Pursuant to the termination agreement, our obligation to supply HEMGENIX^® and any minimum purchase commitments under the Genezen CSA terminate once contractually specified batches have been supplied. In connection with the termination, the Company wrote down the remaining carrying value of the intangible asset for the favorable supply terms under the Genezen CSA. The net effect of these items was recorded in Other expense, and did not have a material impact on the Company’s results of operations for the three and six months ended June 30, 2026.

6Long-term debt

The total principal outstanding as of ~~March~~June 3130, 2026 under the Company's amended venture debt loan facility with Hercules Capital, Inc. (the “2025 Amended Facility”) was $50.0 million. The amortized cost, including interest due presented as part of Accrued expenses and other current liabilities, was $50.35 million as of ~~March~~June 3130, 2026, compared to $50.1 million as of December 31, 2025, and is recorded net of discount and debt issuance costs.

The foreign currency loss on the loan facility in the three and six months ended ~~March~~June 3130, 2026 was $10.5 million and $1.6 million, respectively, compared to a foreign currency gain of $24.13 million and $6.4 million, respectively, during the same ~~period~~periods in 2025. Interest expense during the three and six months ended ~~March~~June 3130, 2026 was $1.4 million and $2.8 million, respectively, compared to $1.8 million and $3.6 million, respectively, during the same ~~period~~periods in 2025.

During the six months ended June 30, 2026, there were no material changes to the terms, available borrowing tranches, covenants, or collateral arrangements of the 2025 Amended Facility as described in Note 11, “Long-term debt” to the consolidated financial statements included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2025. As of June 30, 2026, the Company was in compliance with all applicable covenants under the facility.

In July 2026, subsequent to the balance sheet date, the Company entered into an amendment to its loan facility with Hercules Capital, Inc. (as amended, the “2026 Amended Facility”). The amendment extended the period during which the Company may draw the $100.0 million term loan tranche from June 15, 2027 to September 30, 2027. Except as provided for in the amendment, the terms of borrowing under the 2026 Amended Facility remained unchanged.

During the three months ended March 31, 2026, there were no material changes to the terms, available borrowing tranches, covenants, or collateral arrangements of the 2025 Amended Facility as described in Note 11, “Long-term debt” to the consolidated financial statements included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2025. As of March 31, 2026, the Company was in compliance with all applicable covenants under the facility.

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7Royalty Financing Agreement

During the ~~three~~six months ended ~~March~~June 3130, 2026, there were no material changes to the terms of the royalty purchase agreement entered into in May 2023 (the “Royalty Financing Agreement”) with HemB SPV, L.P. (the “Purchaser”), as described in Note 12, “Royalty Financing Agreement” within the consolidated financial statements included in the Company’s Annual Report for the year ended December 31, 2025.

The following table presents the changes in the liability from ~~royalty~~the ~~financing~~Royalty ~~agreement~~Financing Agreement for the period from December 31, 2025 to ~~March~~June 3130, 2026:


	Amount of liability
	(in thousands)
Balance as of December 31, 2025 (includes $5.5 million presented as "Accrued expenses and other current liabilities")	$	478,722
Royalty payments to the Purchaser		(59,~~523~~195)
Interest expense for the period		1225,~~621~~584
Balance as of March 31, 2026 (includes $3.6 million presented as "Accrued expenses and other current liabilities")	$	485,820
Balance as of June 30, 2026 (includes $5.8 million presented as "Accrued expenses and other current liabilities")	$	495,111

The Company records the debt at amortized cost using the effective interest rate method based on projected cash flows. As of March 31, 2026, the effective interest rate is expected to be within a range of 10.5% to 12.0% per annum (March 31, 2025: 12.0% to 13.5%). Interest expense recorded in connection with the agreement was $12.6 million and $13.3 million for the three months ended March 31, 2026 and 2025, respectively.

The Company records the debt at amortized cost using the effective interest method based on projected cash flows. As of June 30, 2026, the effective interest rate is expected to be within a range of 10.0% to 11.5% per annum (June 30, 2025: 12.0% to 13.5%). Interest expense recorded in connection with the Royalty Financing Agreement was $13.0 million and $13.8 million for the three months ended June 30, 2026 and 2025, respectively, and $25.6 million and $27.1 million for the six months ended June 30, 2026 and 2025, respectively.

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8Shareholders’ equity

In June 2026, the Company received net proceeds of $242.7 million, after deducting underwriting discounts and commissions and other offering expenses payable by the Company, through a follow-on public offering of 5.7 million ordinary shares, at a public offering price of $45.50 per ordinary share.

In June 2026, the Company's shareholders approved an amendment to the Articles of Association increasing the Company's authorized share capital from 80.0 million ordinary shares to 100.0 million ordinary shares with a nominal value of €0.05 per share.

~~8Share~~9Share-based compensation

The Company’s share-based compensation plans include the amended and restated 2014 Share Incentive Plan (as amended, the “2014 Plan”) and inducement grants under Rule 5653(c)(4) of the Nasdaq Global Select Market with terms similar to the 2014 Plan (together the “2014 Plans”). At the annual general meeting of shareholders in June 2026, the Company’s shareholders authorized an additional 350,000 ordinary shares for issuance under the 2014 Plan. As of ~~March~~June 3130, 2026, a total of 2,~~571~~815,~~395~~628 ordinary shares remain available for issuance under the 2014 Plan.

In June 2018, the Company’s shareholders adopted and approved the uniQure N.V. Employee Stock Purchase Plan (as amended, the “ESPP”) allowing the Company to issue up to 150,000 ordinary shares. The ESPP is intended to qualify under Section 423 of the Internal Revenue Code of 1986, as amended. Under the ESPP, employees are eligible to purchase ordinary shares through payroll deductions, subject to any plan limitations. The purchase price of the ordinary shares on each purchase date is equal to the lower of: (i) 85% of the closing market price on the offering date or (ii) 85% of the closing market price on the purchase date.

2014 Plans and ESPP

Share-based compensation expense recognized by classification included in the Unaudited Consolidated Statements of Operations and Comprehensive Loss in relation to the 2014 Plans and the ESPP for the periods indicated below was as follows:


	Three months ended ~~March~~June 3130,				Six months ended June 30,
	2026		2025		2026		2025
	(in thousands)				(in thousands)
Research and development expenses		1,~~977~~888		2,~~463~~758		3,865		5,221
Selling, general and administrative expenses		3,~~113~~128		12,~~947~~326		6,241		4,273
Total	$	5,~~090~~016	$	45,~~410~~084	$	10,106	$	9,494

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Share-based compensation expense recognized by award type for the 2014 Plans as well as the ESPP was as follows:


	Three months ended ~~March~~June 3130,				Six months ended June 30,
	2026		2025		2026		2025
	(in thousands)				(in thousands)
Award type/ESPP
Share options	$	1,~~807~~861	$	1,~~637~~662	$	3,668	$	3,299
Restricted share units		3,~~206~~255		2,~~213~~547		6,461		4,760
Performance share units		62(108)		~~560~~875		(46)		1,435
ESPP		158		—		23		—
Total	$	5,~~090~~016	$	45,~~410~~084	$	10,106	$	9,494

~~As of March 31, 2026, the unrecognized share-based compensation expense related to unvested awards under the 2014 Plans were:~~13

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As of June 30, 2026, the unrecognized share-based compensation expense related to unvested awards under the 2014 Plans was:


	Unrecognized		Weighted average
	share-based		remaining
	compensation		period for
	expense		recognition
	(in thousands)		(in years)
Award type
Share options	$	14,~~881~~464	2.8857
Restricted share units		2725,~~279~~886	2.4017
Performance share units		21427	0.0887
Total	$	4240,~~181~~776	2.5730

The Company satisfies the exercise of share options and vesting of Restricted Share Units (“RSUs”) and Performance Share Units (“PSUs”) through newly issued ordinary shares.

Share options

Share options are priced on the date of grant and, except for certain grants made to non-executive directors, vest over a period of four years. The first 25% of each grant vests one year after the initial grant date and the remainder vests in equal quarterly installments over years two, three and four. Certain grants to non-executive directors vest in full after one year. Any options that vest must be exercised by the tenth anniversary of the initial grant date.

The following table summarizes option activity under the 2014 Plans for the ~~three~~six months ended ~~March~~June 3130, 2026:


	Options
	Number of ordinary shares		Weighted average exercise price

Outstanding at December 31, 2025	4,531,991		17.04
Granted	~~771~~869,~~900~~760	$	911.5348
Forfeited	—(12,451)	$	—9.58
Expired	(1116,~~466~~186)	$	37.0084
Exercised	(25410,~~308~~849)	$	1516.2460
Outstanding at March 31, 2026	5,267,117	$	15.90
Outstanding at June 30, 2026	4,962,265	$	16.05
Thereof, fully vested, and exercisable on March 31, 2026	2,825,796	$	20.06
Thereof, fully vested, and exercisable on June 30, 2026	2,769,675	$	19.77
Thereof, outstanding and expected to vest after March 31, 2026	2,441,321	$	11.09
Thereof, outstanding and expected to vest after June 30, 2026	2,192,590	$	11.35
Outstanding and expected to vest after December 31, 2025	1,999,315	$	11.81


Total weighted average grant date fair value of options issued during the period (in $ millions)				$	46.94

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The fair value of each option issued is estimated at the respective grant date using the Hull & White option pricing model with the following weighted-average assumptions:


	Three months ended ~~March~~June 3130,		Six months ended June 30,
	2026	2025	2026	2025
Assumptions
Expected volatility	95.0%	80.0%	95%	70% - 80%
Expected terms	10 years	10 years	10 years	10 years
Risk-free interest rate	4.268% ~~- 4.3%~~	4.464%	4.21% - 4.68%	4.36% - 4.64%
Expected dividend yield	0.0%	0.0%	0.0%	0.0%

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RSUs

The following table summarizes the RSU activity for the ~~three~~six months ended ~~March~~June 3130, 2026:


	RSUs
		Weighted average
	Number of	grant-date fair
	ordinary shares	value
Non-vested at December 31, 2025	2,395,420	$	12.15
Granted	~~997~~1,~~100~~112,250	$	1012.7736
Vested	(~~657~~886,~~285~~521)	$	1112.3329
Forfeited	(43113,~~468~~350)	$	1516.7222
Non-vested at March 31, 2026	2,691,767	$	11.78
Non-vested at June 30, 2026	2,507,799	$	12.01

Total weighted average grant date fair value of RSUs granted during the period (in $ millions)		$	1013.78

RSUs are measured at grant date based on the market price of the Company’s ordinary shares and, except for certain grants made to non-executive directors, vest over a three-year period. RSUs granted to non-executive directors vest one year after the date of grant.

PSUs

The Company granted PSUs to certain employees in 2024 and 2026, that, with respect to the 2024 PSUs, ~~will~~would behave been earned upon the Board of Directors’ assessment of the level of achievement of an agreed upon performance ~~targets~~target, and, with respect to the 2026 PSUs, will be earned upon Company management’s assessment of the level of achievement of agreed upon performance targets, subject, in each case, to the grantee’s continued employment. The fair value of PSUs granted in 2024 and 2026 was determined on the grant date by reference to the market price of the Company’s ordinary shares.

The Company recognizes the compensation cost related to these grants to the extent it considers achievement of the milestones to be probable. As of ~~March~~June 3130, 2026, ~~the~~certain ~~PSUs granted in~~ 2026 PSUs were assessed as ~~not~~ probable of achieving the requisite performance ~~conditions in accordance with ASC 718, Compensation-Stock Compensation; therefore, no stock-based compensation expense has been recognized~~condition.

~~The~~As ~~following~~of ~~table~~June ~~summarizes~~30, 2026, the ~~PSU~~Company ~~activity~~determined that the performance target for the ~~three~~outstanding ~~months~~2024 ~~ended~~PSUs ~~March~~were ~~31,~~not ~~2026:~~met within the contractually defined performance period and were consequently forfeited.


	PSUs
		Weighted average
	Number of	grant-date fair
	ordinary shares	value
Non-vested at December 31, 2025	22,500	$	15.40
Granted	—	$	—
Vested	—	$	—
Forfeited	—	$	—
Non-vested at March 31, 2026	22,500	$	15.40

The following table summarizes the PSU activity for the six months ended June 30, 2026:


	PSUs
		Weighted average
	Number of	grant-date fair
	ordinary shares	value
Non-vested at December 31, 2025	22,500	$	15.40
Granted	28,750	$	22.40
Vested	—	$	—
Forfeited	(22,500)	$	15.40
Non-vested at June 30, 2026	28,750	$	22.40

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ESPP

During the ~~three~~six months ended ~~March~~June 3130, 2026, 13,939 ordinary shares were issued under the ESPP compared to nil ordinary shares issued during the same period in 2025. As of ~~March~~June 3130, 2026, 72,773 ordinary shares remain available for issuance under the ESPP compared to a total of 86,712 ordinary shares as of ~~March~~June 3130, 2025.

~~9 Segment reporting~~15

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10	Segment reporting

The Company is advancing a pipeline of innovative gene therapies seeking to deliver to patients suffering from rare and other devastating diseases single treatments with potentially curative results. The Company manages the operations related to these research and development activities within one operating segment because they rely on a common set of infrastructure, resources and technology of the products and production processes, types of customers, distribution methods and regulatory environment.

The leadership team is identified as the Chief Operating Decision Maker (“CODM”).

The CODM allocates resources to research projects and clinical candidates based on scientific data as well as quantitative and qualitative expected risk-adjusted returns on investment. The CODM uses segment operating loss to monitor that cash operating losses remain within the approved budget.

The accounting policies of the operating segment are the same as those described in the summary of significant accounting policies.


	Three months ended ~~March~~June 3130,				Six months ended June 30,
	2026		2025		2026		2025
	(in thousands)				(in thousands)
Revenue	$	35,~~562~~841	$	15,~~567~~262	$	9,403	$	6,829
Less:
Employee related expenses		(16,~~786~~960)		(14,~~216~~525)		(33,746)		(28,741)
Laboratory and development expenses		(1417,~~761~~387)		(15,~~967~~593)		(32,148)		(31,560)
Professional fees		(41,~~507~~756)		(23,~~057~~124)		(6,263)		(5,181)
Information technology system costs		(1,659)		(1,001)
Information technology system costs(2)		(1,427)		(838)		(3,086)		(1,839)
Facility expenses		(1,~~547~~437)		(1,~~667~~704)		(2,984)		(3,371)
Other segment items(1)		(3,513)		(969)
Other segment items(1) (2)		(2,511)		(1,804)		(6,024)		(2,773)
Segment operating loss		(3935,~~211~~637)		(3432,~~310~~326)		(74,848)		(66,636)
Reconciliation
Depreciation and amortization expense		(24,~~956~~907)		(43,~~527~~630)		(7,863)		(8,157)
Share-based compensation expense		(5,~~090~~016)		(45,~~410~~084)		(10,106)		(9,494)
Fair value gain / (loss) - contingent consideration		1,338		(1,216)
Fair value (loss) / gain - contingent consideration		(1,256)		(2,254)		82		(3,470)
Fair value gain - liability related to pre-funded warrants		3,769		—
Fair value loss - Liability related to pre-funded warrants		(15,970)		—		(12,201)		—
Foreign currency (losses) / gains, net		(21,~~294~~730)		718,~~172~~638		(4,024)		25,810
Interest income		5,~~229~~051		43,~~127~~524		10,280		7,651
Interest expense - Royalty Financing Agreement		(12,~~621~~963)		(13,~~309~~770)		(25,584)		(27,079)
Interest expense - Hercules loan facility		(1,~~410~~403)		(1,~~800~~821)		(2,813)		(3,621)
Other reconciling items		~~199~~(5,387)		~~5,132~~(571)		(5,188)		4,561
Consolidated loss before income tax expense	$	(5379,~~047~~218)	$	(4337,~~141~~294)	$	(132,265)	$	(80,435)

(1)

Other segment items included in segment operating loss include costs related to intellectual property and insurance offset by income related to payments received from European authorities to subsidize the Company’s research and development in the Netherlands.

(2)

The comparative amounts for the three and six months ended June 30, 2025 have been adjusted to reflect the level of detail at which the CODM reviews segment operating expenses in 2026, with information technology system costs presented as a separate line item and consumables included within other segment items for all periods presented.

1416

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1011 Other investments and concentration of credit risk

The following table summarizes the Company’s other investments as of ~~March~~June 3130, 2026 and December 31, 2025:


	~~March~~June 3130,		December 31,
	2026		2025
	(in thousands)
Carrying amount of investments valued under measurement alternative	$	15,280	$	15,615
Carrying amount of equity investments	$	15,143	$	15,615
Convertible note receivable		1415,~~870~~124		14,622
Total	$	30,~~150~~267	$	30,237

The Company ~~did~~holds ~~not~~investments ~~recognize~~in ~~any~~non-publicly ~~upward~~traded ~~adjustments,~~equity ~~downward~~securities ~~adjustments~~without readily determinable fair values. These investments are accounted for at cost, orless ~~impairments~~impairment ~~relating~~and adjusted to ~~investments~~fair ~~carried~~value ~~under~~if there are observable price changes in orderly transactions for identical or similar investments using the measurement alternative ~~during~~in ~~the~~accordance ~~three~~with ~~months~~ASC ~~ended~~321, ~~March~~Investments 31– Equity Securities. The carrying value of these investments as of June 30, 2026, and December 31, 2025 was $15.1 million and $15.6 million, respectively, and are presented within Other Investments in the Company’s Consolidated Balance Sheets.

The Company did not recognize any upward adjustments, downward adjustments, or impairments relating to investments carried under the measurement alternative during the three and six months ended June 30, 2026, and 2025.

Genezen

In 2024, as part of the Company’s divestment of its commercial manufacturing operations to Genezen ~~Holdings Inc. and its subsidiary Genezen MA~~, ~~Inc. (together “Genezen”),~~ the Company entered into avarious ~~commercial~~service ~~supply~~agreements ~~agreement~~including (the “Genezen CSA~~”), as well as a development and other manufacturing agreement, with Genezen~~. In ~~The~~April ~~Company recorded a balance of prepaid expenses of $12.3 million and $13.9 million as of March 31,~~ 2026 ~~and December 31~~, ~~2025, respectively, for services that~~ the Company ~~expects~~entered tointo ~~receive~~an ~~from~~agreement ~~Genezen.~~to Interminate ~~addition, as of March 31, 2026,~~ the ~~Company~~Genezen ~~holds a convertible note receivable of $14~~CSA.9 ~~million~~Pursuant ~~which~~to ~~is due in September 2029 and for which Genezen is~~ the ~~counterparty~~termination ~~(December 31~~agreement, ~~2025: $14.6 million). These balances represent a concentration of credit risk, and~~ the Company’s ~~maximum~~obligation ~~exposure~~ to ~~loss~~supply isHEMGENIX® ~~the~~and ~~carrying~~any ~~value~~minimum ofpurchase ~~these~~commitments ~~assets.~~under Nothe ~~expected~~Genezen ~~credit~~CSA ~~losses~~terminate ~~were~~once ~~recognized~~contractually ~~during~~specified ~~the~~batches ~~three~~have ~~months~~been ~~ended March 31, 2026 or 2025~~supplied.

~~CSL~~The ~~Behring~~Company’s prepayments for services to be received amounted to $5.7 million as of June 30, 2026, compared to $13.9 million as of December 31, 2025. In addition, as of June 30, 2026, the Company holds a convertible note receivable of $15.1 million which is due in September 2029 and for which Genezen is the counterparty (December 31, 2025: $14.6 million).

AsThese balances represent a concentration of ~~March~~credit 31risk, ~~2026~~ and ~~December~~the ~~31,~~Company’s ~~2025,~~maximum ~~accounts~~exposure ~~receivable~~to ~~from~~loss ~~CSL~~is ~~Behring~~the ~~LLC~~carrying ~~(“CSL~~value ~~Behring”)~~of ~~totaled~~these $3assets.6 ~~million~~No ~~and~~expected ~~$5.9~~credit ~~million,~~losses ~~respectively,~~were inrecognized ~~each~~during ~~case~~the ~~related~~three toand ~~royalty~~six ~~revenue~~months ended June 30, 2026 or 2025.

CSL Behring

As of June 30, 2026 and December 31, 2025, accounts receivable from CSL Behring LLC (“CSL Behring”) totaled $5.8 million and $5.9 million, respectively, in each case related to royalty revenue which is included in License Revenue in the accompanying statements of operations and comprehensive loss.

11 Income taxes

The Company recorded a deferred tax expense of $0.5 million related to its U.S. operations during the three months ended March 31, 2026 ($0.5 million for the three months ended March 31, 2025).

~~The~~12 ~~effective~~Income income tax rate of 0.9% during the three months ended March 31, 2026 (three months ended March 31, 2025: 1.1%) is substantially lower than the enacted rate of 25.8% in the Netherlands as the Company recorded a valuation allowance against its net deferred tax assets in the Netherlands and a partial valuation allowance against its net deferred tax assets in France.taxes

The Company recorded a deferred tax expense of $1.8 million and $2.3 million related to its U.S. operations during the three and six months ended June 30, 2026 ($0.4 million and $0.9 million for the three and six months ended June 30, 2025).

The effective income tax rate of 2.3% and 1.8% during the three and six months ended June 30, 2026 (three and six months ended June 30, 2025: 1.1% and 1.1%, respectively) is substantially lower than the enacted rate of 25.8% in the Netherlands as the Company recorded a valuation allowance against its net deferred tax assets in the Netherlands and a partial valuation allowance against its net deferred tax assets in France, among various other items which impacted the effective rate.

12Basic and diluted loss per share

Diluted loss per ordinary share is calculated by adjusting the weighted average number of ordinary shares outstanding, assuming conversion of all potentially dilutive ordinary shares. As the Company incurred a loss in the three months ended March 31, 2026 and March 31, 2025, all potentially dilutive ordinary shares would have an antidilutive effect, if converted, and thus have been excluded from the computation of diluted loss per share for the three months ended March 31, 2026 and March 31, 2025. The ordinary shares are presented without giving effect to the application of the treasury method or exercise prices that would be above the share price as of March 31, 2026 and March 31, 2025, respectively.17

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13Basic and diluted loss per share

Diluted loss per ordinary share is calculated by adjusting the weighted average number of ordinary shares outstanding, assuming conversion of all potentially dilutive ordinary shares. As the Company incurred a loss in the three and six months ended June 30, 2026, all potentially dilutive ordinary shares would have an antidilutive effect, if converted, and thus have been excluded from the computation of diluted loss per share for the three and six months ended June 30, 2026. The ordinary shares are presented without giving effect to the application of the treasury method or exercise prices that would be above the share price as of June 30, 2026 and June 30, 2025, respectively.

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The potentially dilutive ordinary shares are summarized below:


	Three months ended June 30,	~~March 31,~~					Six months ended June 30,
	~~ ~~2026	~~2026~~		~~ ~~2025	~~2025~~		2026		2025
Anti-dilutive ordinary share equivalents
Stock options under 2014 Plans	$	$4,962,265	~~5,267,117~~	$	$5,321,862	~~5,283,396~~	$	4,962,265	$	5,321,862
Non-vested RSUs and PSUs		2,536,549	~~2,714,267~~		2,442,969	~~2,242,926~~		2,536,549		2,442,969
Pre-Funded Warrants		526,316	~~526,316~~		—	—		526,316		—
ESPP		13,939	~~13,936~~		—	—		13,939		—
Total anti-dilutive ordinary share equivalents		8,039,069	~~8,521,636~~		7,764,831	~~7,526,322~~		8,039,069		7,764,831

~~13Commitments~~14Commitments and contingencies

In the course of its business, the Company enters as a licensee into contracts with other parties regarding the development and marketing of its pipeline products. Among other payment obligations, the Company is obligated to pay royalties to the licensors based on future sales levels and milestone payments whenever specified development, regulatory and commercial milestones are met. As both future sales levels and the timing and achievement of milestones are uncertain, the financial effect of these agreements cannot be reliably estimated. The Company also has obligations to make future payments that become due and payable upon the collection of milestone payments from CSL Behring. The achievement and timing of these milestones isare not fixed or determinable.

As of March 31, 2026, the remaining minimum purchase commitments in connection with the Genezen CSA amount to $16.2 million, with $13.3 million to be paid within the next 12 months. As of March 31, 2026, CSL Behring’s minimum purchase commitments to the Company over the next 12 months, in connection with the development and commercial supply agreement between the Company and CSL Behring (the “CSL Behring CSA”), amount to $16.4 million, of which $16.2 million relates to such minimum purchase commitments to Genezen. In April 2026, the Company entered into agreements with Genezen and CSL Behring to terminate the Genezen CSA and the CSL Behring CSA. Refer to Note 14, “Subsequent events”.

Legal Proceedings

On February 10, 2026, a class action complaint captioned Christopher Scocco v. uniQure N.V., et al., Case No. 1:26-cv-01124, was filed against the Company, certain of its executive officers and another party (collectively, “Defendants”) in the United States District Court for the Southern District of New York. The complaint ~~purports~~purported to assert claims pursuant to Sections 10(b) and 20(a) of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), and Rule 10b-5 promulgated thereunder, on behalf of a putative class of investors who purchased or otherwise acquired the Company’s ordinary shares between September 24, 2025 and October 31, 2025 (the “putative Class Period”). ~~Plaintiff~~The ~~seeks~~plaintiff sought to recover damages allegedly caused by purported false and misleading statements and omissions with respect to the Company’s Phase I/II study of AMT-130 and the timing of the potential ~~BLA~~Biologics License Application filing for AMT-130. ~~The Company intends to vigorously defend against the claims in this action.~~

On April 13, 2026, the court appointed the Oklahoma Firefighters Pension and Retirement System as lead plaintiff (the “Lead Plaintiff”).

AtOn ~~each~~July ~~reporting~~15, ~~date~~2026, the ~~Company~~Lead ~~evaluates~~Plaintiff ~~whether~~filed oran ~~not~~amended acomplaint. ~~potential~~Like ~~loss~~the ~~amount~~earlier-filed orcomplaint, athe ~~potential~~amended ~~range~~complaint purports to assert claims pursuant to Sections 10(b) and 20(a) of ~~loss~~the isExchange ~~probable~~Act and ~~reasonably~~Rule ~~estimable~~10b-5, ~~under~~on ~~the~~behalf ~~provisions~~of a putative class of investors who purchased or otherwise acquired the ~~authoritative~~Company’s ~~guidance~~ordinary ~~that~~shares ~~addresses~~during ~~accounting~~the ~~for~~putative ~~contingencies~~Class Period. AtLead ~~this~~Plaintiff ~~time,~~seeks ~~the~~unspecified ~~Company~~damages isallegedly ~~unable~~caused by purported false and misleading statements and omissions with respect to ~~predict~~the import and viability of data from the ~~outcome~~Company’s Phase I/II study of AMT-130 and available pathways to regulatory approval. Defendants’ motion to dismiss the ~~class~~amended ~~action~~complaint ~~litigation~~is orcurrently ~~reasonably~~due ~~estimate~~September a14, ~~range~~2026. ofManagement ~~possible~~intends ~~losses~~to vigorously defend against the claims in this action.

At each reporting date, the Company evaluates whether or not a potential loss amount or a potential range of loss is probable and reasonably estimable under the provisions of the authoritative guidance that addresses accounting for contingencies. The Company has not recorded any liability for any portion of this matter because the Company believes that liability is not probable and reasonably estimable at this time.

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~~14Subsequent~~15Subsequent events

~~Termination~~In ~~agreements~~July 2026, subsequent to the balance sheet date, the Company entered into an amendment to its loan facility with ~~CSL~~Hercules ~~Behring~~Capital, ~~and~~Inc. ~~Genezen~~(as amended, the “2026 Amended Facility”). The amendment extended the period during which the Company may draw the $100.0 million term loan tranche from June 15, 2027 to September 30, 2027. Except as provided for in the amendment, the terms of borrowing under the 2026 Amended Facility remained unchanged.

Since the Company’s July 2024 divestiture of its commercial manufacturing activities to Genezen, the Company had been sourcing etranacogene dezaparvovec-drlb (“HEMGENIX®”) under the Genezen CSA. The Company entered into the Genezen CSA to enable it to meet its obligations to supply HEMGENIX® in accordance with the June 2020 commercialization and license agreement with CSL Behring (the “CSL Behring Agreement”) (refer to Note 10, “Other Investments and concentration of credit risk,” and Note 13, “Commitments and contingencies”) until such time that the HEMGENIX® manufacturing technology had been transferred to CSL Behring or a contract manufacturing organization designated by CSL Behring. The Company, since July 2024, had served as an agent in the sale of HEMGENIX® to CSL Behring as title to HEMGENIX® drug product supply had been directly passing from Genezen to CSL Behring. In April 2026, the Company entered into agreements with CSL Behring and Genezen that provide for i) the termination of its obligation to supply HEMGENIX® and any minimum purchase commitments under the Genezen CSA, once the contractually specified batches have been supplied to CSL Behring, which the Company expects to occur in mid-2026, and ii) the designation of Genezen as CSL Behring’s contract manufacturing organization. In addition, the CSL Behring Agreement was amended to terminate certain manufacturing-related terms associated with both the CSL Behring CSA and the Genezen CSA, as well as the Company’s development support that CSL Behring could request from time to time with respect to HEMGENIX®. All other terms of the CSL Behring Agreement remain in full force and effect.

The Company expects that by the time the contractually specified batches have been supplied to CSL Behring, it will i) have fully amortized the carrying amount of the intangible asset recorded with respect to the favorable supply terms under the Genezen CSA of $7.3 million as of March 31, 2026, and ii) have fully released the firm purchase commitment liability with a March 31, 2026 carrying amount of $5.8 million.19

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Item 2.Management’s Discussion and Analysis of Financial Condition and Results of Operations

The following discussion of our results of operations and financial condition should be read in conjunction with our unaudited consolidated financial statements and the accompanying notes thereto and other disclosures included in this Quarterly Report on Form 10-Q, including the disclosures under Part II, Item 1A “Risk Factors,” and our audited financial information and the notes thereto included in our Annual Report on Form 10-K (the “Annual Report”). Our unaudited consolidated financial statements have been prepared in accordance with generally accepted accounting principles in the United States (“U.S. GAAP”) and unless otherwise indicated are presented in United States (“U.S.”) dollars.

Overview

We are a leader in the field of gene therapy, seeking to deliver to patients suffering from rare and other devastating diseases single treatments with potentially curative results. We are advancing a focused pipeline of innovative gene therapies, including our clinical candidates for the treatment of Huntington’s disease, refractory mesial temporal lobe epilepsy (“MTLE”), and Fabry disease.

Business Developments

Financing

In June 2026, we received net proceeds of $242.7 million, after deducting underwriting discounts and commissions and other offering expenses payable by us, through a follow-on public offering of 5.7 million ordinary shares, at a public offering price of $45.50 per ordinary share.

Hercules Loan Amendment

In July 2026, we entered into an amendment to the $175.0 million senior secured term loan facility (the “2026 Amended Facility”) with Hercules Capital, Inc. (“Hercules”). The 2026 Amended Facility, among other things, extends the period we can draw the $100.0 million term loan tranche from June 2027 to September 2027. Except as provided for in the amendment, the terms of borrowing under the 2026 Amended Facility otherwise remain unchanged.

Recent Product Candidate Developments

Huntington’s disease program (AMT-130)

AMT-130 is our novel gene therapy candidate for the treatment of Huntington’s disease, which utilizes our proprietary, gene-silencing miQURE^® platform and incorporates a miRNA, specifically designed to silence the huntingtin gene and the potentially highly toxic exon 1 protein fragment.

We are currently conducting Phase I/II clinical trials of AMT-130 in the U.S. and Europe. We completed the enrollment of all 26 patients in the first two cohorts of our U.S. study in March 2022 and the enrollment of 13 patients in the two cohorts of our European study in June 2023. In 2025, we completed enrollment of all 12 patients in the third cohort, and we treated six patients with the high-dose of AMT-130 in a fourth cohort to evaluate the safety and efficacy of AMT-130 in patients with lower baseline striatal volumes compared to previous cohorts in the U.S. Phase I/II study.

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Data from Phase I/II Clinical Studies

In September 2025, we announced positive topline data from the three-year analysis of cohorts one and two of the ongoing Phase I/II studies of AMT-130 for the treatment of Huntington’s disease. We analyzed clinical outcomes for 29 patients treated with AMT-130 (n=17 high-dose; n=12 low-dose) of which 12 patients per dose group had attained 36 months of follow-up and were evaluated at that time point. Outcomes for each dose group were compared to a propensity score-matched external control drawn from the Enroll-HD natural history data set (n=940 for high-dose; n=626 for low-dose).

Topline 36-month efficacy results for patients receiving high-dose AMT-130 were as follows (data cutoff as of June 30, 2025):

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●

A statistically significant 75% slowing of disease progression as measured by composite Unified Huntington’s Disease Rating Scale (“cUHDRS”) (p=0.003), which met the primary endpoint of the study. Treated patients had a mean change in cUHDRS from baseline of -0.38 compared to a change of -1.52 for patients in the propensity score-matched external control.

●

A statistically significant 60% slowing of disease progression as measured by Total Function Capacity (“TFC”) (p=0.033), which met a key secondary endpoint of the study. Treated patients had a mean change in TFC from baseline of -0.36 compared to a change of -0.88 for patients in the propensity score-matched external control.

●

Favorable trends in other secondary endpoint measures of motor and cognitive function, including Symbol Digit Modalities Test (“SDMT”), Stroop Word Reading Test (“SWRT”) and Total Motor Score (“TMS”).

o

An 88% slowing of disease progression as measured by SDMT (p=0.057), with a mean change in SDMT from baseline of -0.44 compared to a change of -3.73 for patients in the propensity score-matched external control.

o

A 113% slowing of disease progression as measured by SWRT (nominal p=0.002), with a mean change in SWRT from baseline of 0.88 compared to a change of -6.98 for patients in the propensity score-matched external control.

o

A 59% slowing of disease progression as measured by TMS (nominal p=0.174), with a mean change in TMS from baseline of 2.01 compared to a change of 4.88 for patients in the propensity score-matched external control.

A mean reduction from baseline in cerebrospinal neurofilament light protein (“CSF NfL”) of -8.2% was observed at 36 months in the high-dose of AMT-130 of the Phase I/II studies. CSF NfL is a well-characterized, supportive biomarker of neurodegeneration. Elevation in CSF NfL has been shown to be strongly associated with greater clinical severity of Huntington’s disease.

We believe that the consistently favorable results in functional, motor and cognitive endpoints at 36 months observed in the high-dose group, compared to the variable trends observed in the low-dose group, reflect a dose-dependent response to AMT-130.

Various other supportive analyses of the results from the AMT-130 high-dose treatment group, including those using a propensity score-weighted external control and comparisons to the TRACK-HD and PREDICT-HD datasets, were consistent with the primary analysis.

AMT-130 was generally well-tolerated in the Phase I/II studies, with a manageable safety profile at both doses. ~~The~~There have been five drug-related serious adverse events (“SAEs”) reported across all cohorts, and the most common adverse events in the treatment groups were related to the administration procedure.

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Regulatory Update

From November 2024 through April 2025, we held three Type B meetings with the U.S. Food and Drug Administration (“FDA”). As part of these interactions, the FDA agreed that data from the ongoing Phase I/II studies, compared to a natural history external control, may serve as the primary basis of a Biologics License Application (“BLA”) submission under the FDA’s accelerated approval pathway. The FDA also agreed that cUHDRS may be used as an intermediate clinical endpoint and reductions in CSF NfL may serve as supportive evidence of therapeutic benefit in the application for such accelerated approval.

In October 2025, we met with the FDA at a pre-BLA meeting to discuss the application for AMT-130. In December 2025, we announced that in the final meeting minutes, the FDA conveyed that data submitted from the Phase I/II studies of AMT-130 are currently unlikely to provide the primary evidence to support a BLA submission.

In January 2026, we met with the FDA at a Type A meeting to discuss AMT-130. In March 2026, following receipt of the final meeting minutes from the Type A meeting, we announced that the FDA stated that it cannot agree that data from the Phase I/II studies, compared to an external control, are sufficient to provide the primary evidence of effectiveness required to support a marketing application for AMT-130. The FDA strongly recommended we conduct a prospective, randomized, double-blind, sham surgery-controlled study.

WeFrom ~~intend to~~November ~~continue~~2024 ~~engaging~~through April 2025, we held three Type B meetings with the ~~FDA~~U.S. Food and ~~have~~Drug ~~scheduled~~Administration (the “FDA”). As part of these interactions, the FDA agreed that data from the ongoing Phase I/II studies, compared to a ~~Type~~natural Bhistory ~~meeting~~external ~~with~~control, may serve as the ~~agency~~primary inbasis of a Biologics License Application (“BLA”) submission under the ~~second~~FDA’s ~~quarter~~accelerated approval pathway. The FDA also agreed that cUHDRS may be used as an intermediate clinical endpoint and reductions in CSF NfL may serve as supportive evidence of ~~2026~~therapeutic benefit in the application for such accelerated approval.

In ~~April~~October ~~2026~~2025, we ~~announced~~met ~~that,~~with ~~following~~the ~~feedback~~FDA ~~from~~at a pre-~~submission~~BLA meeting ~~with~~to discuss the ~~United~~application ~~Kingdom’s~~for ~~(“UK”)~~AMT-130. ~~Medicines~~In ~~and~~December ~~Healthcare~~2025, ~~products~~we ~~Regulatory~~announced ~~Agency~~that ~~(“MHRA”)~~in ~~regarding~~the ~~AMT-130~~final meeting minutes, wethe ~~plan~~FDA toconveyed ~~submit~~that adata UKsubmitted ~~Marketing~~from ~~Authorization~~the ~~Application~~Phase ~~for~~I/II studies of AMT-130 inwere currently unlikely to provide the ~~third~~primary ~~quarter~~evidence ofto ~~2026~~support a BLA submission.

~~Temporal lobe epilepsy program (AMT-260)~~21

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In January 2026, we met with the FDA at a Type A meeting to discuss AMT-130. In March 2026, following receipt of the final meeting minutes from the Type A meeting, we announced that the FDA stated that it could not agree that data from the Phase I/II studies, compared to an external control, were sufficient to provide the primary evidence of effectiveness required to support a marketing application for AMT-130. The FDA strongly recommended we conduct a prospective, randomized, double-blind, sham surgery-controlled study.

WeIn ~~are~~March ~~conducting a Phase I/IIa clinical trial~~2026, ~~GenTLE,~~we ofheld ~~AMT-260 for the treatment of MTLE in the U.S. GenTLE consists of two parts. The first part is~~ a ~~multicenter,~~successful ~~open~~pre-~~label~~submission ~~trial~~meeting with ~~two~~the ~~dosing~~United ~~cohorts~~Kingdom’s of(“UK”) ~~six~~Medicines ~~patients~~and ~~each~~Healthcare toproducts ~~assess~~Regulatory ~~safety,~~Agency ~~tolerability,~~(“MHRA”) ~~and~~regarding ~~initial efficacy of~~ AMT-~~260~~130, inand ~~patients~~the ~~with~~regulatory ~~refractory~~submission ~~MTLE. The second part~~ is ~~expected~~progressing toas beplanned afor ~~randomized,~~the ~~controlled~~third ~~trial~~quarter ~~to generate proof~~ of ~~concept data~~2026.

In ~~September~~June ~~2025~~2026, we ~~completed~~held ~~enrollment~~a ofType B meeting with the ~~first~~FDA. ~~three~~Official ~~patients~~meeting minutes received in July 2026 confirmed that we and the ~~first~~FDA ~~cohort~~reached ~~administering~~alignment that a BLA submission under the accelerated approval pathway for AMT-~~260~~130, tobased ~~patients~~on ~~with~~the ~~lesions~~existing inclinical data, is reasonable. In addition, the ~~non-dominant~~FDA ~~hemisphere~~seeks ofto align on the ~~brain.~~confirmatory ~~Following~~study adesign ~~review~~prior byto the ~~independent~~BLA ~~data~~submission, ~~monitoring~~including ~~committee~~consideration ~~(“IDMC”)~~of a randomized standard-of-care control design instead of a sham procedure. The FDA also stated that, wein ~~expanded~~accordance with the ~~first~~FDA’s ~~cohort~~draft ~~into~~public ~~MTLE~~guidance infor accelerated approvals, the ~~dominant~~confirmatory ~~hemisphere~~study ~~and~~should ~~initiated~~be feasible to conduct within a ~~second~~reasonable ~~cohort.~~timeline Weand ~~completed~~be ~~enrollment~~well ofunderway, ~~six~~and ~~patients~~potentially ~~into~~fully enrolled, at the ~~first~~time ~~cohort~~of inaccelerated ~~2025~~approval. WeDiscussions ~~also~~with ~~initiated~~the ~~enrollment~~FDA ofto aalign ~~second~~on ~~cohort~~the inconfirmatory ~~2025~~study design and analysis are underway, ~~which~~and iswe ~~expected~~expect to ~~include~~submit ana ~~additional~~BLA ~~six~~in ~~patients~~the third quarter of 2026.

~~Fabry~~Temporal ~~disease~~lobe epilepsy program (AMT-~~191~~260)

We are conducting a Phase I/IIIIa clinical trial, GenTLE, of AMT-~~191~~260 for the treatment of ~~Fabry~~MTLE ~~disease~~in the U.S. ~~The~~GenTLE is a multicenter, open-label ~~clinical~~ trial ~~consists~~with oftwo ~~three~~dosing ~~dose-ranging~~ cohorts of ~~three~~at orleast ~~more~~six patients each to assess safety, tolerability, and initial efficacy of AMT-~~191~~260 in patients with ~~Fabry~~refractory ~~disease~~MTLE.

In ~~February~~September ~~2026~~2025, we ~~announced~~completed ~~updated~~enrollment ~~preliminary~~of ~~data~~the ~~from~~first ~~the~~three ~~Phase~~patients ~~I/II~~in ~~study~~the offirst cohort administering AMT-~~191~~260 ~~for~~to ~~Fabry~~patients ~~disease~~with lesions in the non-dominant hemisphere of the brain. ~~Updated~~ ~~preliminary~~Following ~~safety~~a ~~and~~review ~~exploratory~~by ~~efficacy~~the independent data ~~included~~monitoring ~~the~~committee ~~following~~(“IDMC”), ~~with~~we expanded the first cohort into MTLE in the dominant hemisphere and initiated a ~~data~~second ~~cutoff~~cohort. ~~date~~We ascompleted enrollment of ~~January~~six 8,patients ~~2026:~~into the first cohort in 2025. We also initiated enrollment of a second cohort in 2025.

●In ~~Dose-dependent~~June ~~elevations~~2026, ~~were~~we ~~observed~~announced ~~across~~preliminary 11data ~~patients~~on inthe ~~three~~first ~~dose~~cohort ~~levels~~in ~~with~~GenTLE. ~~α-Gal~~As Aof ~~activity~~the ~~ranging~~May ~~from~~29, ~~0.34-~~2026 todata ~~82.2-fold~~cutoff ~~above~~date, ~~mean~~three ~~normal~~of ~~range~~six ~~(1.38-8.66~~patients ~~nmol;~~in ~~mean~~the ~~normal~~first, oflow-dose ~~3.57~~cohort ~~nmol~~(1x1012 gc/mL) atachieved ~~the~~meaningful ~~lowest~~reductions ~~dose,~~in ~~1.6-~~disabling toseizures ~~312.52-fold~~during atmonths ~~the~~four ~~mid~~through ~~dose,~~six ~~and~~of ~~27.7~~follow-up, ranging from a 79% to ~~223~~100% decline from baseline.~~7-fold~~ atThe ~~the~~remaining ~~highest~~three patients in the low-dose. ~~These~~cohort ~~increases~~experienced ~~were~~variable ~~durable~~changes ~~for~~in ~~the~~disabling ~~measured~~seizures ~~time~~during ~~period~~months ~~ranging~~four ~~from~~through ~~the~~six ~~longest~~of follow-up, ~~period~~ranging offrom ~~more~~a ~~than~~33% decrease to a ~~year~~36% inincrease acompared ~~treated~~to ~~patient~~baseline. ~~(high-dose~~As ~~cohort)~~of the June 19, 2026, there have been no SAEs related to AMT-260 or the ~~shortest~~surgical ~~follow-up~~procedure ~~period~~reported. ofAll ~~four-months~~reported inadverse aevents ~~treated~~in ~~patient~~the ~~(mid-~~low dose cohort were classified as mild or moderate in severity, with the most common adverse event being headache (N=2). No immunosuppression was required.

● Six of 11 dosed patients were withdrawn from enzyme replacement therapy (“ERT”).

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● Plasma lyso-Gb3 levels were stable post-dose across all dose cohorts, regardless of ERT status through the cutoff date.

AsFabry ofdisease ~~February~~program ~~18, 2026, all 11 dosed patients were withdrawn from ERT.~~(AMT-191)

~~AMT-191~~We ~~continued~~are toconducting ~~show~~ a ~~manageable~~Phase ~~safety~~I/II ~~profile.~~clinical Notrial ~~SAEs~~of ~~related to~~ AMT-191 ~~were~~for ~~observed at~~ the ~~4x1013 gc/kg~~treatment ~~and~~of ~~2x1013 gc/kg~~Fabry ~~doses~~disease. No ~~additional~~The ~~SAEs were observed at the 6x1013 gc/kg dose beyond the five previously reported in September 2025 in two patients. Per protocol~~multicenter, ~~additional~~open-label ~~dosing~~clinical intrial ~~the~~consists ~~mid-~~of ~~and~~three ~~high-~~dose-ranging cohorts ~~has~~of ~~been~~three ~~paused~~or ~~pending~~more ~~further~~patients ~~evaluation~~each ~~following~~to ~~asymptomatic~~assess ~~Grade~~safety, 3tolerability, ~~liver~~and ~~enzyme~~efficacy ~~elevations~~of ~~observed~~AMT-191 in ~~two~~ patients inwith ~~the~~Fabry ~~mid-dose cohort, which were confirmed dose-limiting toxicity~~disease.

~~Amyotrophic~~In ~~Lateral~~June ~~Sclerosis~~2026, (we presented updated preliminary data from the Phase I/II study of AMT-~~162)~~191 for Fabry disease. The data, based on a March 15, 2026 data cutoff date, included patient follow-up ranging from three months to more than 18 months and consisted of the following:

~~EPISOD1~~● ~~is a Phase I/II multi~~Dose-~~center,~~dependent ~~open-label~~elevations ~~trial~~were ofobserved ~~AMT-162~~across ~~for~~11 ~~the~~patients ~~treatment~~in ofthree ~~amyotrophic~~dose ~~lateral~~levels ~~sclerosis~~with ~~caused~~α-Gal byA ~~mutations~~activity inranging ~~superoxide~~from ~~dismutase~~ 1 ~~(“SOD1~~.0-~~ALS”)~~ into ~~the U~~16.S.2-fold Inabove ~~September~~mean ~~2025,~~normal werange ~~voluntarily~~(1.38-8.66 ~~paused~~nmol; ~~enrollment~~mean innormal ~~EPISOD1 upon the recommendation~~ of ~~the~~3.57 ~~IDMC~~nmol) ~~following~~at ~~a review of available preliminary data related to~~ the ~~safety~~lowest ~~and efficacy of AMT-162 in the context of a~~ dose ~~limiting toxicity~~, ~~which~~14.5- ~~resulted in a SAE determined~~ to ~~be related to AMT~~229.6-~~162,~~fold ~~that~~at ~~was observed in one patient in~~ the ~~second~~mid ~~cohort.~~dose, ~~Following review of the preliminary efficacy~~ and ~~safety~~58.7- ~~data generated from EPISOD1, we have decided~~ to ~~discontinue development of AMT-162~~143.6-fold Weat ~~will continue to collect safety data from~~ the ~~five~~highest ~~patients dosed in EPISOD1, consistent with applicable safety and regulatory requirements~~dose.

● All 11 dosed patients were withdrawn from enzyme replacement therapy (“ERT”).

~~CSL~~● ~~Behring~~Plasma ~~Collaboration~~lyso-Gb3 levels were stable post-dose across all dose cohorts, regardless of ERT status through the cutoff date.

In June 2020, we entered into a commercialization and license agreement with CSL Behring LLC (the “CSL Behring Agreement”) pursuant to which CSL Behring LLC (“CSL Behring”) received exclusive global rights to HEMGENIX®.22

We and CSL Behring also entered into a development and commercial supply agreement, pursuant to which, among other things, we agreed to supply the product to CSL Behring until such time that these capabilities are transferred to CSL Behring or its designated contract manufacturing organization (the “CSL Behring CSA”). CSL Behring in September 2022 informed us about their intent to transfer contract manufacturing to a designated third party. In July 2024 as part of our divestment of our commercial manufacturing operations to Genezen Holdings Inc. and its subsidiary Genezen MA, Inc. (together “Genezen”), we entered into a commercial supply agreement to transfer the manufacturing and supply activities to Genezen such that Genezen manufactures and supplies CSL Behring’s commercial demand for HEMGENIX® on our behalf (the “Genezen CSA”).

In April 2026, we entered into agreements with CSL Behring and Genezen that provide for (i) the termination of our obligation to supply HEMGENIX® and any minimum purchase commitments under the Genezen CSA, once the contractually specified batches have been supplied to CSL Behring, which we expect to occur in mid-2026, and (ii) the designation of Genezen as CSL Behring’s contract manufacturing organization. In addition, the CSL Behring Agreement was amended to terminate certain manufacturing-related terms associated with both the CSL Behring CSA and the Genezen CSA, as well as our development support that CSL Behring could request from time to time with respect to HEMGENIX®. All other terms of the CSL Behring Agreement remain in full force and effect.

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AMT-191 continued to show a manageable safety profile. No SAEs related to AMT-191 were observed at the 4x10¹³ gc/kg (“mid-dose”) and 2x10¹³ gc/kg (“low-dose”) doses. No additional SAEs were observed at the 6x10¹³ gc/kg (“high-dose”) dose beyond those previously reported in September 2025 in two patients.

Per protocol, additional dosing in the mid- and high-dose cohorts has been paused pending further evaluation following asymptomatic Grade 3 liver enzyme elevations observed in two patients in the mid-dose cohort, which were confirmed dose-limiting toxicity. These elevations resolved as of the end of May 2026 following a course of immunosuppression as per the study protocol.

Financial Overview

Key components of our results of operations include the following:


	Three months ended ~~March~~June 3130,				Six months ended June 30,
	2026		2025		2026		2025
	(in thousands)				(in thousands)
Total revenues	$	35,~~562~~841	$	15,~~567~~262	$	9,403	$	6,829
Cost of license revenues		(~~219~~350)		(~~197~~656)		(569)		(853)
Research and development expenses		(2933,~~176~~964)		(3635,~~140~~383)		(63,140)		(71,523)
Selling, general and administrative expenses		(2017,~~068~~367)		(1013,~~908~~500)		(37,435)		(24,408)
Net loss		(5381,~~535~~059)		(4337,~~637~~719)		(134,594)		(81,356)

As of ~~March~~June 3130, 2026 and December 31, 2025, we had cash and cash equivalents and investment securities of $~~586~~810.63 million and $622.5 million, respectively. We had a net loss of $5381.51 million and $134.6 million in the three and six months ended ~~March~~June 3130, 2026, compared to a net loss of $4337.67 million and $81.4 million for the same ~~period~~periods in 2025. As of ~~March~~June 3130, 2026 and December 31, 2025, we had accumulated deficits of $1,~~382~~463.5 million and $1,328.9 million, respectively. See “Results of Operations” below for a discussion of the detailed components and analysis of the amounts above.

Critical Accounting Policies and Estimates

In preparing our unaudited consolidated financial statements in accordance with U.S. GAAP and pursuant to the rules and regulations promulgated by the Securities and Exchange Commission (the “SEC”) we make assumptions, judgments and estimates that can have a significant impact on our net loss and affect the reported amounts of certain assets, liabilities, revenue and expenses, and related disclosures. We base our assumptions, judgments and estimates on historical experience and various other factors that we believe to be reasonable under the circumstances, the results of which form the basis for making judgments about the carrying values of assets and liabilities that are not clear from other sources. Actual results may differ from these estimates under different assumptions or conditions. In making estimates and judgments, management employs critical accounting policies. A summary of our critical accounting policies, as well as a discussion of our critical accounting estimates, are presented in our Annual Report. There were no material changes to our critical accounting policies during the ~~three~~six months ended ~~March~~June 3130, 2026.

Research and development expenses

We expense research and development (“R&D”) expenses as incurred. R&D expenses include costs which relate to our primary activities of biopharmaceutical research and development. Our R&D expenses generally consist of costs incurred for the development of our target candidates, which include:

●

employee-related expenses, including salaries, benefits, travel and share-based compensation expense;

●

costs incurred for laboratory research, preclinical and nonclinical studies, clinical trials, statistical analysis and report writing, and regulatory compliance costs incurred with clinical research organizations and other third-party vendors;

●

costs incurred to conduct consistency and comparability studies;

●

costs incurred for the development and improvement of our manufacturing processes and methods;

●

costs associated with research activities for enabling technology platforms;

●

costs associated with the rendering of collaboration services;

●

payments related to identifiable intangible assets without an alternative future use;

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●

payments to our licensors for milestones that have been achieved related to our product candidates;

● facilities, depreciation, and other expenses, which include direct and allocated expenses for rent and maintenance of facilities, insurance, and other supplies; and,

●

changes in the fair value of liabilities recorded in relation to the acquisition of uniQure France SAS.

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Our R&D expenses may vary substantially from period to period based on the timing of our research and development activities, including manufacturing campaigns, regulatory submissions, and enrollment of patients in clinical trials. The successful development of our product candidates is highly uncertain. Estimating the nature, timing, or cost of the development of any of our product candidates involves considerable judgment due to numerous risks and uncertainties associated with developing gene therapies, including the uncertainty of:

●

the scope, rate of progress and expense of our research and development activities;

●

clinical trial protocols, speed of enrollment and resulting data;

● the effectiveness and safety of our product candidates; and,

●

the timing of regulatory approvals.

A change in the outcome of any of these variables with respect to our product candidates that we may develop could mean a significant change in the expenses and timing associated with the development of such product candidates.

Selling, general and administrative expenses

Our selling, general and administrative expenses consist principally of employee, office, consulting, legal and other professional and administrative expenses. We incurred expenses associated with operating as a public company, including expenses for personnel, legal, accounting and audit fees, board of directors’ costs, directors'’ and officers'’ liability insurance premiums, Nasdaq listing fees, expenses related to investor relations and fees related to business development and maintaining our patent and license portfolio.

Other items, net

Our other income generally consists of payments received to subsidize our research and development efforts and income from the subleasing of our Amsterdam facility and our Lexington, MA research and development facility.

In 2024, as part of the divestment of our commercial manufacturing operations to Genezen Holdings Inc. and its subsidiary Genezen MA, Inc. (together “Genezen”), we entered into various service agreements, including a commercial supply agreement (the “Genezen CSA”). In April 2026, we entered into an agreement to terminate the Genezen CSA. Pursuant to the termination agreement, our obligation to supply HEMGENIX^® and any minimum purchase commitments under the Genezen CSA terminate once contractually specified batches have been supplied.

Our other expenses consist of costs incurred under the Genezen CSA. These include costs related to the purchase of HEMGENIX^® from Genezen, net of income from the sales of HEMGENIX^® to CSL Behring LLC (“CSL Behring”), amortization of the intangible asset recorded with respect to the favorable supply terms under the Genezen CSA, and release of liabilities related to expected net losses associated with the remaining minimum purchase commitments under the Genezen CSA.

Additionally, other expenses also consist of expenses we incur in relation to our subleasing income.

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Results of Operations

Comparison of the three months ended June 30, 2026 and 2025

23The following table presents a comparison of our results of operations for the three months ended June 30, 2026 and 2025:


		Three months ended June 30,
		2026			2025			2026 vs 2025

		(in thousands)
License revenues		$	5,841		$	5,262		$	579
Total revenues			5,841			5,262			579
Operating expenses:
Cost of license revenues			(350)			(656)			306
Research and development expenses			(33,964)			(35,383)			1,419
Selling, general and administrative expenses			(17,367)			(13,500)			(3,867)
Total operating expenses			(51,681)			(49,539)			(2,142)
Other income			1,598			2,597			(999)
Other expense			(7,961)			(2,185)			(5,776)
Loss from operations			(52,203)			(43,865)			(8,338)
Non-operating (expense) / income, net			(27,015)			6,571			(33,586)
Net loss before income tax expense		$	(79,218)		$	(37,294)		$	(41,924)
Income tax expense			(1,841)			(425)			(1,416)
Net loss		$	(81,059)		$	(37,719)		$	(43,340)

License revenues

We recognize license revenues from CSL Behring related to royalty payments owed on HEMGENIX^® sales, when earned. For the three months ended June 30, 2026 and 2025, we recognized $5.8 million and $5.3 million of license revenues, respectively.

R&D expense

R&D expenses for the three months ended June 30, 2026 were $34.0 million, compared to $35.4 million for the same period in 2025. Other research and development expenses are separately classified in the table below. These other expenses are not allocated to specific projects, as they are deployed across multiple projects under development.


	Three months ended June 30,
	2026		2025		2026 vs 2025

	(in thousands)
Huntington's disease (AMT-130)	$	6,280	$	10,517	$	(4,237)
Temporal lobe epilepsy (AMT-260)		4,168		1,955		2,213
Amyotrophic lateral sclerosis (AMT-162)		2,503		1,098		1,405
Fabry disease (AMT-191)		2,177		1,255		922
Programs in preclinical development and platform related expenses		1,883		405		1,478
Total direct research and development expenses	$	17,011	$	15,230	$	1,781

Employee and contractor-related expenses		9,111		9,523		(412)
Facility expenses		2,402		3,853		(1,451)
Share-based compensation expense		1,888		2,758		(870)
Information technology costs		966		594		372
Fair value changes related to contingent consideration		1,256		2,254		(998)
Other expenses		1,330		1,171		159
Total other research and development expenses	$	16,953	$	20,153	$	(3,200)

Total research and development expenses	$	33,964	$	35,383	$	(1,419)

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Direct research and development expenses

Huntington’s disease (AMT-130)

In the three months ended June 30, 2026 and 2025, we incurred costs related to the development of AMT-130 of $6.3 million and $10.5 million respectively. The decrease of $4.2 million was primarily related to lower manufacturing process validation costs, which were $0.8 million in the current year period, compared to $4.0 million in the prior year period. Additionally, clinical trials costs decreased in the current year period, partially offset by higher regulatory costs associated with the preparation of BLA and Marketing Authorization Application (“MAA”) submissions for AMT-130 in the United States and the United Kingdom, respectively.

Temporal lobe epilepsy (AMT-260)

In the three months ended June 30, 2026 and June 30, 2025, we incurred costs related to the development of AMT-260 of $4.2 million and $2.0 million, respectively. The current year period included $3.7 million of clinical expenses and $0.3 million of CMC expenses, respectively, compared to $1.7 million and $0.2 million, respectively, in the prior year period.

Amyotrophic Lateral Sclerosis caused by mutations in SOD1 (AMT-162)

In the three months ended June 30, 2026 and June 30, 2025, we incurred costs related to the development of AMT-162 of $2.5 million and $1.1 million, respectively. These costs were primarily related to our Phase I/II clinical trial.

Fabry disease (AMT-191)

In the three months ended June 30, 2026 and June 30, 2025, we incurred costs related to the development of AMT-191 of $2.2 million and $1.3 million, respectively. These costs were primarily related to our Phase I/II trial.

Preclinical programs & platform development

In the three months ended June 30, 2026 and June 30, 2025, we incurred costs related to research and technology projects of $1.9 million and $0.4 million, respectively.

Other research & development expenses

●

We incurred $9.1 million in personnel and contractor-related expenses in the three months ended June 30, 2026, compared to $9.5 million for the same period in 2025;

●

We incurred $2.4 million in operating and depreciation expenses related to our leased facilities in Amsterdam and Lexington, Massachusetts in the three months ended June 30, 2026 compared to $3.9 million in the same period in 2025;

●

We incurred $1.9 million in share-based compensation expenses in the three months ended June 30, 2026, compared to $2.8 million for the same period in 2025. The decrease of $0.9 million was primarily driven by lower costs related to performance stock units during the current year period; and

●

We incurred a $1.3 million loss related to an increase in the fair value of contingent consideration associated with the acquisition of uniQure France SAS in the three months ended June 30, 2026, compared to a $2.3 million loss for the same period in 2025. The $1.0 million favorable change reflects that the prior period included an adjustment to the expected timing of achieving future milestones that did not recur in the current year period.

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Selling, general and administrative expenses

Selling, general and administrative expenses for the three months ended June 30, 2026 were $17.4 million, compared to $13.5 million for the same period in 2025.


	Three months ended June 30,
	2026		2025		2026 vs 2025

	(in thousands)
Employee and contractor-related expenses	$	8,878	$	5,397	$	3,481
Share-based compensation expense		3,128		2,326		802
Professional fees		1,592		3,428		(1,836)
Intellectual property fees		983		281		702
Depreciation and facility costs		508		578		(70)
Information technology costs		389		193		196
Other expenses		1,889		1,297		592
Total selling, general and administrative expenses	$	17,367	$	13,500	$	3,867

●

We incurred $8.9 million in personnel and contractor-related expenses in the three months ended June 30, 2026, compared to $5.4 million in the same period in 2025. The $3.5 million increase was primarily driven by of a higher headcount following employee recruitment in the second half of 2025 to support the potential commercial launches of AMT-130;

●

We incurred $3.1 million in share-based compensation expenses in the three months ended June 30, 2026, compared to $2.3 million in the same period in 2025. The $0.8 million increase was primarily driven by the higher fair value of awards outstanding during the current year period, in comparison with the prior period;

●

We incurred $1.6 million in professional fees in the three months ended June 30, 2026 compared to $3.4 million in the same period in 2025. The $1.8 million decrease was primarily a result of lower costs in connection with the potential commercial launches of AMT-130; and

●

We incurred $1.9 million in other expenses in the three months ended June 30, 2026, compared to $1.3 million in the same period in 2025.

Other income and expense


	Three months ended June 30,
	2026		2025		2026 vs 2025

	(in thousands)
Research and development grants from Dutch authorities	$	1,164	$	1,392	$	(228)
Sublease income, net		337		347		(10)
Supply of HEMGENIX^® to CSL Behring		(7,695)		(1,731)		(5,964)
Other income, net		(169)		404		(573)
Total other items, net	$	(6,363)	$	412	$	(6,775)

In April 2026, we entered into an agreement to terminate the Genezen CSA. In connection with the termination, we recorded a write-down of $5.9 million related to the favorable supply intangible asset within Other expense.

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Other non-operating items, net

Our other non-operating items, net, for the three months ended June 30, 2026 and June 30, 2025 were as follows:


		Three months ended June 30,
		2026			2025			2026 vs 2025

		(in thousands)
Interest income		$	5,051		$	3,524		$	1,527
Interest expense - Royalty Financing Agreement			(12,963)			(13,770)			807
Interest expense - Hercules loan facility			(1,403)			(1,821)			418
Foreign currency (losses) / gains, net			(1,730)			18,638			(20,368)
Changes in fair value of liability related to pre-funded warrants			(15,970)			—			(15,970)
Total non-operating items, net		$	(27,015)		$	6,571		$	(33,586)

We recognize interest income associated with our cash and cash equivalents and investment securities. We recognized $5.1 million in interest income in the three months ended June 30, 2026, compared to $3.5 million in the same period in 2025. The $1.5 million increase was primarily due to higher average balances of our investment securities held during the current year period, compared to the prior year period.

In May 2023, uniQure biopharma B.V. entered into an agreement (the “Royalty Financing Agreement”) with HemB SPV, L.P. to sell certain current and future royalties due to uniQure biopharma B.V. from CSL Behring from the net sales of HEMGENIX^® pursuant to the CSL Behring Agreement. We recognized non-cash interest expenses related to the Royalty Financing Agreement of $13.0 million and $13.8 million in the three months ended June 30, 2026 and 2025, respectively.

We recognized interest expense related to the Hercules loan facility of $1.4 million and $1.8 million in the three months ended June 30, 2026 and June 30, 2025, respectively. The $0.4 million decrease was primarily due to more favorable terms following the amendment of the facility in September 2025, as well as a decrease in market interest rates.

We conduct transactions and hold monetary assets and liabilities denominated in foreign currencies, principally the euro and the U.S. dollar. Monetary balances are remeasured at period-end exchange rates, and resulting foreign currency transaction gains and losses are recorded in the Unaudited Consolidated Statements of Operations and Comprehensive Loss as incurred.

We recognized a net foreign currency loss of $1.7 million during the three months ended June 30, 2026, compared to a net gain of $18.6 million during the same period in 2025. The foreign currency movements relate to our Hercules loan facility, the Royalty Financing Agreement, cash and cash equivalents, investment securities, and intercompany loans within the uniQure group.

In connection with our September 2025 follow-on public offering, we issued pre-funded warrants that are classified as a liability and measured at fair value each reporting period. We recognized a $16.0 million loss in the three months ended June 30, 2026, reflecting an increase in the fair value of this liability, compared to nil for the same period in 2025.

Income tax expense

We recognized $1.8 million of deferred tax expense in the three months ended June 30, 2026, and $0.4 million of deferred tax expense for the same period in 2025.

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Results of Operations

Comparison of the ~~three~~six months ended ~~March~~June 3130, 2026 and 2025

The following table presents a comparison of our results of operations for the ~~three~~six months ended ~~March~~June 3130, 2026 and 2025:


		Six months ended June 30,
		2026			2025		2026 vs 2025

		(in thousands)
Total revenues		$	9,403		$	6,829	$	2,574
Operating expenses:
Cost of license revenues			(569)			(853)		284
Research and development expenses			(63,140)			(71,523)		8,383
Selling, general and administrative expenses			(37,435)			(24,408)		(13,027)
Total operating expenses			(101,144)			(96,784)		(4,360)
Other income			3,230			10,903		(7,673)
Other expense			(9,412)			(4,144)		(5,268)
Loss from operations			(97,923)			(83,196)		(14,727)
Non-operating (expense) / income, net			(34,342)			2,761		(37,103)
Loss before income tax expense		$	(132,265)		$	(80,435)		(51,830)
Income tax expense			(2,329)			(921)		(1,408)
Net loss		$	(134,594)		$	(81,356)	$	(53,238)


	Three months ended March 31,
	2026		2025		2026 vs 2025
	(in thousands)
License revenues	$	3,562	$	1,567	$	1,995
Total revenues		3,562		1,567		1,995
Operating expenses:
Cost of license revenues		(219)		(197)		(22)
Research and development expenses		(29,176)		(36,140)		6,964
Selling, general and administrative expenses		(20,068)		(10,908)		(9,160)
Total operating expenses		(49,463)		(47,245)		(2,218)
Other income		1,632		8,306		(6,674)
Other expense		(1,451)		(1,959)		508
Loss from operations		(45,720)		(39,331)		(6,389)
Non-operating expense, net		(7,327)		(3,810)		(3,517)
Net loss before income tax expense	$	(53,047)	$	(43,141)	$	(9,906)
Income tax expense		(488)		(496)		8
Net loss	$	(53,535)	$	(43,637)	$	(9,898)

License revenues

We recognize license revenues from CSL Behring related to royalty payments owed on HEMGENIX® sales, when earned. For the three months ended March 31, 2026 and 2025, we recognized $3.6 million and $1.6 million of license revenues, respectively.

We recognize license revenues from CSL Behring related to royalty payments owed on HEMGENIX^® sales, when earned. For the six months ended June 30, 2026 and 2025, we recognized $9.4 million and $6.8 million of license revenues, respectively.

R&D expense

R&D expenses for the three months ended March 31, 2026 were $29.2 million, compared to $36.1 million for the same period in 2025. Other research and development expenses are separately classified in the table below. These other expenses are not allocated to specific projects, as they are deployed across multiple projects under development.


	Three months ended March 31,
	2026		2025		2026 vs 2025
	(in thousands)
Huntington's disease (AMT-130)	$	7,980	$	8,230	$	(250)
Temporal lobe epilepsy (AMT-260)		2,827		2,075		752
Fabry disease (AMT-191)		1,539		2,187		(648)
Amyotrophic lateral sclerosis (AMT-162)		894		1,831		(937)
Programs in preclinical development and platform related expenses		1,134		1,280		(146)
Total direct research and development expenses	$	14,374	$	15,603	$	(1,229)

Employee and contractor-related expenses		9,060		10,159		(1,099)
Facility expenses		3,619		4,550		(931)
Share-based compensation expense		1,977		2,463		(486)
Fair value changes related to contingent consideration		(1,338)		1,216		(2,554)
Other expenses		1,484		2,149		(665)
Total other research and development expenses	$	14,802	$	20,537	$	(5,735)

Total research and development expenses	$	29,176	$	36,140	$	(6,964)

R&D expenses for the six months ended June 30, 2026 were $63.1 million, compared to $71.5 million for the same period in 2025. Other research and development expenses are separately classified in the table below. These other expenses are not allocated to specific projects, as they are deployed across multiple projects under development.


		Six months ended June 30,
		2026			2025			2026 vs 2025

		(in thousands)
Huntington's disease (AMT-130)		$	14,260		$	18,747		$	(4,487)
Temporal lobe epilepsy (AMT-260)			6,995			4,030			2,965
Fabry disease (AMT-191)			3,716			3,442			274
Amyotrophic lateral sclerosis (AMT-162)			3,397			2,929			468
Programs in preclinical development and platform related expenses			3,017			1,685			1,332
Total direct research and development expenses		$	31,385		$	30,833		$	552

Employee and contractor-related expenses			18,171			19,682			(1,511)
Facility expenses			6,021			8,403			(2,382)
Share-based compensation expense			3,865			5,221			(1,356)
Information technology costs			2,056			1,276			780
Fair value changes related to contingent consideration			(82)			3,470			(3,552)
Other expenses			1,724			2,638			(914)
Total other research and development expenses		$	31,755		$	40,690		$	(8,935)

Total research and development expenses		$	63,140		$	71,523		$	(8,383)

Table of Contents

Direct research and development expenses

Huntington’s disease (AMT-130)

In the three months ended March 31, 2026 and 2025, we incurred costs of $8.0 million and $8.2 million respectively, primarily related to clinical trials and Chemistry, Manufacturing, and Controls (“CMC”) development. We incurred costs of $4.5 million and $3.0 million related to clinical trials and CMC development, respectively, compared to costs of $5.7 million and $2.0 million in the comparative period.

In the six months ended June 30, 2026 and 2025, we incurred costs related to the development of AMT-130 of $14.3 million and $18.7 million respectively. The decrease of $4.5 million was primarily driven by lower costs related to clinical trials of $9.0 million in the current period, compared to $11.2 million in the prior year period. Additionally, manufacturing process validation costs were decreased in the current year period, partially offset by higher regulatory costs associated with the preparation of BLA and MAA submissions for AMT-130 in the United States and the United Kingdom, respectively.

Temporal lobe epilepsy (AMT-260)

In the three months ended March 31, 2026 and March 31, 2025 we incurred costs of $2.8 million and $2.1 million respectively, for the development of AMT-260. We incurred costs of $2.6 million and $0.2 million related to clinical trials and CMC development, respectively, compared to costs of $1.6 million and $0.4 million in the comparative period.

In the six months ended June 30, 2026 and June 30, 2025, we incurred costs related to the development of AMT-260 of $7.0 million and $4.0 million, respectively. The current period included costs of $6.3 million related to clinical trials and $0.5 million related to CMC development, compared to $3.3 million and $0.7 million, respectively, in the prior year period.

Fabry disease (AMT-191)

In the three months ended March 31, 2026 and March 31, 2025, we incurred costs of $1.5 million and $2.2 million, respectively, related to our development of AMT-191, primarily in relation to our Phae I/II trial.

In the six months ended June 30, 2026 and June 30, 2025, we incurred costs related to the development of AMT-191 of $3.7 million and $3.4 million, respectively. These costs primarily related to the Phase I/II trial.

Amyotrophic Lateral Sclerosis caused by mutations in SOD1 (AMT-162)

~~In the three months ended March 31, 2026 and March 31, 2025, we incurred $0.9 million and $1.8 million of expenses, respectively, primarily in relation to our Phase I/II clinical trial.~~

In the six months ended June 30, 2026 and June 30, 2025, we incurred costs related to the development of AMT-162 of $3.4 million and $2.9 million, respectively. These costs primarily related to the Phase I/II trial.

Preclinical programs & platform development

In the three months ended March 31, 2026 and March 31, 2025, we incurred $1.1 million and $1.3 million of costs, respectively, related to activities associated with product candidates for various other research programs and technology innovation projects.

In the six months ended June 30, 2026 and June 30, 2025, we incurred costs related to research and technology projects of $3.0 million and $1.7 million, respectively.

Other research & development expenses

● We incurred $9.1 million in personnel and contractor-related expenses in the three months ended March 31, 2026, compared to $10.2 million for the same period in 2025. The decrease was due to a decrease in personnel-related expenses, as well as a decrease in severance costs and reorganization expenses related to the divestment of our commercial manufacturing activities in July 2024, incurred in the first quarter of 2025;

● We incurred $318.62 million in ~~operating~~personnel and ~~depreciation~~contractor-related expenses ~~related to our leased facilities~~ in ~~Amsterdam and Lexington, Massachusetts in~~ the ~~three~~six months ended ~~March~~June 3130, 2026, compared to $419.67 million infor the same period in 2025. The decrease of $1.05 million was primarily ~~driven~~due ~~by a revision~~ to ~~the~~lower ~~estimated~~personnel-related ~~useful~~expenses ~~lives of certain assets, resulting~~ in ~~higher depreciation recognized during~~ the ~~three months ended March 31, 2025, compared with the~~ current year period;

● We ~~recognized~~incurred a $16.30 million ~~gain~~ in ~~the~~operating ~~three~~and ~~months~~depreciation ~~ended~~expenses ~~March 31, 2026,~~ related to aour ~~decrease~~leased facilities in ~~the~~Amsterdam ~~fair~~and ~~value~~Lexington, ofMassachusetts ~~contingent~~in ~~consideration associated with~~ the ~~acquisition~~six ofmonths ~~uniQure~~ended ~~France~~June ~~SAS~~30, 2026 compared to a $18.24 million ~~loss~~in ~~for~~ the same period in 2025. The decrease of $2.4 million was primarily ~~related~~driven toby ~~changes~~a revision to the ~~discount~~estimated ~~rate~~useful ~~and~~lives ~~expected~~of ~~timing~~certain ofassets, ~~future~~resulting ~~achievement~~in ofhigher depreciation recognized during the ~~related~~prior ~~remaining~~year ~~milestones~~period;

● We incurred $23.09 million in share-based compensation expenses in the ~~three~~six months ended ~~March~~June 3130, 2026, compared to $2.5.2 million for the same period in 2025;. ~~and,~~The decrease of $1.3 million was primarily driven by lower costs related to performance stock units during the current year period;

● We incurred a $10.51 million gain related to a decrease in the fair value of ~~other~~contingent ~~expenses~~consideration ~~for~~associated with the ~~three~~acquisition of uniQure France SAS in the six months ended ~~March~~June 3130, 2026, compared to a $23.15 million loss for the same period in 2025. The $3.6 million favorable change was primarily driven by movements in the discount rate and the timing of future milestone achievements; and

25● We incurred $1.7 million of other expenses for the six months ended June 30, 2026, compared to $2.6 million for the same period in 2025.

~~Table of Contents~~

Selling, general and administrative expenses

Selling, general and administrative expenses for the three months ended March 31, 2026 were $20.1 million, compared to $10.9 million for the same period in 2025.


	Three months ended March 31,
	2026		2025		2026 vs 2025
	(in thousands)
Employee and contractor-related expenses	$	9,855	$	5,485	$	4,370
Professional fees		3,655		1,903		1,752
Share-based compensation expense		3,113		1,947		1,166
Intellectual property fees		872		283		589
Depreciation and facility costs		337		380		(43)
Information technology costs		520		281		239
Other expenses		1,716		629		1,087
Total selling, general and administrative expenses	$	20,068	$	10,908	$	9,160

●Selling, Wegeneral ~~incurred $9.9 million in personnel~~ and ~~contractor-related~~administrative expenses infor the ~~three~~six months ended ~~March~~June 3130, 2026 were $37.4 million, compared to $524.54 million infor the same period in 2025. ~~The increase in 2026 was primarily the result of a higher number of employees recruited in the second half of 2025 in support of a potential commercial launch of AMT-130;~~

● We incurred $3.7 million in professional fees in the three months ended March 31, 2026 compared to $1.9 million in the same period in 2025. The increase was related to fees incurred in relation to legal and commercial services;

● We incurred $3.1 million in share-based compensation expenses in the three months ended March 31, 2026, compared to $1.9 million in the same period in 2025. The increase was primarily the result of a higher number of grants outstanding in the three months ended March 31, 2026, in comparison with the prior period; and,

● We incurred $1.7 million in other expenses in the three months ended March 31, 2026, compared to $0.6 million in the same period in 2025.

Table of Contents


		Six months ended June 30,
		2026			2025			2026 vs 2025

		(in thousands)
Employee and contractor-related expenses		$	18,733		$	10,882		$	7,851
Share-based compensation expense			6,241			4,273			1,968
Professional fees			5,247			5,331			(84)
Intellectual property fees			1,855			564			1,291
Information technology costs			909			474			435
Depreciation and facility costs			845			958			(113)
Other expenses			3,605			1,926			1,679
Total selling, general and administrative expenses		$	37,435		$	24,408		$	13,027

●

We incurred $18.7 million in personnel and contractor-related expenses in the six months ended June 30, 2026, compared to $10.9 million in the same period in 2025. The $7.8 million increase was primarily driven by higher headcount following employee recruitment in the second half of 2025 to support the potential commercial launches of AMT-130;

●

We incurred $5.2 million in professional fees in the six months ended June 30, 2026 compared to $5.3 million in the same period in 2025;

●

We incurred $6.2 million in share-based compensation expenses in the six months ended June 30, 2026, compared to $4.3 million in the same period in 2025. The $1.9 million increase was primarily driven by the higher fair value of awards outstanding during the current year period, in comparison with the prior period;

●

We incurred $1.9 million in intellectual property fees in the six months ended June 30, 2026 compared to $0.6 million in the same period in 2025. The $1.3 million increase was primarily driven by higher registration and professional fees; and

●

We incurred $3.6 million in other expenses in the six months ended June 30, 2026, compared to $1.9 million in the same period in 2025.

Other income and expense


	~~Three~~Six months ended ~~March~~June 3130,
	2026		2025		2026 vs 2025
	(in thousands)
Research and development grants from Dutch authorities	$	~~958~~2,122	$	13,~~719~~111	$	(~~761~~989)
Sublease income / (expense), net		269		24		245
Sublease income, net		606		371		235
Sale of critical reagents to Genezen	$	—	$	6,000	$	(6,000)
Supply of HEMGENIX® to CSL Behring		(18,~~185~~880)		(13,~~525~~256)		~~340~~(5,624)
Other income, net		~~139~~(30)		~~129~~533		10(563)
Total other items, net	$	~~181~~(6,182)	$	6,~~347~~759	$	(612,~~166~~941)

~~Other~~In ~~non-operating~~April ~~items~~2026, ~~net~~we entered into an agreement to terminate the Genezen CSA. In connection with the termination, we recorded a write-down of $5.9 million related to the favorable supply intangible asset in Other expense.

Our other non-operating items, net, for the three months ended March 31, 2026 and March 31, 2025 were as follows:


	Three months ended March 31,
	2026		2025		2026 vs 2025
	(in thousands)
Interest income	$	5,229	$	4,127	$	1,102
Interest expense - Royalty Financing Agreement		(12,621)		(13,309)		688
Interest expense - Hercules debt facility		(1,410)		(1,800)		390
Foreign currency (losses) / gains, net		(2,294)		7,172		(9,466)
Changes in fair value of liability related to pre-funded warrants		3,769		—		3,769
Total non-operating items, net	$	(7,327)	$	(3,810)	$	(3,517)

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WeOther ~~recognize~~non-operating ~~interest income associated with our cash and cash equivalents and investment securities. We recognized $5.2 million in interest income in the three months ended March 31~~items, 2026, compared to $4.1 million in the same period in the prior year. Our interest income increased by $1.1 million, primarily due to increases in amounts of our investment securities during the three months ended March 31, 2026, compared to the same period in the prior year.net

In May 2023, uniQure biopharma B.V. entered into an agreement (the “Royalty Financing Agreement”) with HemB SPV, L.P. to sell certain current and future royalties due to uniQure biopharma B.V. from CSL Behring from the net sales of HEMGENIX® pursuant to the CSL Behring Agreement. We recognized non-cash interest expenses related to the Royalty Financing Agreement of $12.6 million and $13.3 million in the three months ended March 31, 2026 and 2025, respectively.

WeOur ~~recognized~~other ~~interest~~non-operating ~~expense~~items, ofnet, ~~$1.4~~for ~~million and $1.8 million in~~ the ~~three~~six months ended ~~March~~June 3130, 2026, and ~~March~~June 3130, 2025, ~~respectively,~~were ~~related to the Hercules loan facility. Our interest expense decreased by $0.4 million, primarily due to improved terms following the September 2025 amendment~~ as ~~well as a decrease in market interest rates.~~ follows:

We recognized a net foreign currency loss, related to our borrowings from Hercules, the Royalty Financing Agreement and our cash and cash equivalents and investment securities as well as loans between entities within the uniQure group, of $2.3 million during the three months ended March 31, 2026, compared to a net gain of $7.2 million during the same period in 2025.


	Six months ended June 30,
	2026		2025		2026 vs 2025

	(in thousands)
Interest income	$	10,280	$	7,651	$	2,629
Interest expense - Royalty Financing Agreement		(25,584)		(27,079)		1,495
Interest expense - Hercules loan facility		(2,814)		(3,621)		807
Foreign currency (losses) / gains, net		(4,024)		25,810		(29,834)
Changes in fair value of liability related to pre-funded warrants		(12,201)		—		(12,201)
Total Other non-operating items, net	$	(34,342)	$	2,761	$	(37,103)

We issued pre-funded warrants as part of our September 2025 follow-on public offering. We recognize changes related to a liability recorded in relation to this issuance at fair value. We recognized a $3.8 million gain in the three months ended March 31, 2026 related to a decrease in the fair value of the liability related to pre-funded warrants, compared to nil for the same period in 2025.

We recognize interest income associated with our cash and cash equivalents and investment securities. We recognized $10.3 million in interest income in the six months ended June 30, 2026, compared to $7.7 million in the same period in 2025. The $2.6 million increase was primarily due to higher average balances of investment securities held during the current year period, compared to the prior year period.

We recognized interest expense related to the Hercules loan facility of $2.8 million in the six months ended June 30, 2026, compared to $3.6 million in the same period in 2025. The $0.8 million decrease was primarily due to more favorable terms following the amendment of the facility in September 2025, as well as a decrease in market interest rates.

We recognized a net foreign currency loss of $4.0 million in the six months ended June 30, 2026, compared to a net gain of $25.8 million in the same period in 2025. The foreign currency movements related to our borrowings from Hercules, the Royalty Financing Agreement, cash and cash equivalents, investment securities, and intercompany loans within the uniQure group.

Income tax expense

~~We recognized $0.5 million of deferred tax expense in the three months ended March 31, 2026, and $0.5 million of deferred tax expense for the same period in 2025.~~

27We recognized $2.3 million of deferred tax expense in the six months ended June 30, 2026, compared to $0.9 million in the same period in 2025.

Table of Contents

Financial Position, Liquidity and Capital Resources

As of June 30, 2026, we had cash and cash equivalents, restricted cash and investment securities of $811.9 million. We believe these resources will be sufficient to fund our projected operating expenses into 2030, including costs associated with the commercial launches and confirmatory study of AMT-130, ongoing clinical trials of AMT-130, AMT-191 and AMT-260, and potential investments to advance certain pipeline candidates into late stage development. The amount and timing of our actual expenditures may vary significantly depending on the commencement and design of any new clinical studies that we may conduct. We have based our estimate on assumptions that may prove to be wrong, and we could exhaust our available capital resources sooner than we expect, and as such, we may require additional funding.

AsThis ofincludes ~~March~~up ~~31,~~to ~~2026, we had cash and cash equivalents, restricted cash and investment securities of~~ $~~588~~100.10 million. Wein ~~believe~~additional ~~our~~availability ~~cash~~under ~~and cash equivalents, restricted cash, and investment securities will be sufficient to fund~~ our ~~projected~~Hercules ~~operating~~senior ~~expenses~~secured ~~into~~term ~~the~~loan ~~second~~facility, ~~half~~which ofbecomes ~~2029.~~accessible ~~Such~~upon ~~operating~~BLA ~~expenses~~approval ~~include~~for ~~expenses to fund the ongoing clinical trials of~~ AMT-130, ~~AMT-191~~prior ~~and~~to ~~AMT-260~~September 30, as2027, ~~well~~subject asto ~~the~~confirmatory ~~potential~~trial ~~investment~~requirements ofremaining aon ~~material~~track. ~~portion~~Additional offunding ~~the~~could ~~proceeds~~also ~~from~~include ~~our~~a ~~September~~combination ~~2025~~of public ~~follow-on~~equity ~~offering~~offerings, ~~primarily~~collaborations, ~~into~~strategic ~~advancing~~alliances, ~~certain~~licensing ofarrangements ~~our~~or ~~clinical~~marketing ~~candidates into late state development. The amount~~ and ~~timing~~distribution ofarrangements, ~~our~~which ~~actual expenditures~~ may ~~vary~~not ~~significantly~~be ~~depending~~possible. onIf ~~when~~adequate wefunds ~~commence,~~are asnot ~~well~~available asto ~~the~~us ~~design~~on ~~of,~~acceptable ~~any~~terms ~~Phase~~when ~~III~~we ~~trials~~need ~~for AMT-130~~them, ~~AMT-191, and AMT-260 that~~ we may ~~conduct.~~be Weunable ~~have based our estimate on assumptions that may prove~~ to bepursue ~~wrong,~~further ~~and~~development weof ~~could exhaust~~ our ~~available~~clinical ~~capital~~product ~~resources sooner than we expect, and as such, we may require additional funding~~candidates.

If the BLA for AMT-130 is approved prior to June 15, 2027, provided that confirmatory trials to the extent and in the manner required to support full approval (if applicable) remain ongoing or are being planned, we could draw down up to $100.0 million under our senior secured term loan facility with Hercules. Additional funding could also include a combination of public equity offerings, collaborations, strategic alliances, licensing arrangements or marketing and distribution arrangements, which may not be possible. If adequate funds are not available to us on acceptable terms when we need them, we may be unable to pursue further development of our clinical product candidates.

Our current material cash requirements include the following contractual and other obligations:

Debt

As of ~~March~~June 3130, 2026, we had an outstanding loan amount owed to Hercules for an aggregate principal amount of $50.0 million. The loan has an interest-only period until October 1, 2028 and we are contractually required to repay the $50.0 million in equal installments between October 1, 2028 and October 1, 2030. The interest-only period will be extended to October 1, 2029 if the BLA for AMT-130 is approved prior to ~~June~~October 151, ~~2027~~2028. The ~~loan~~interest-only period will be ~~interest-only~~further ~~until~~extended to October 1, 2030 if certain commercial milestones are met prior to ~~December~~March 31, ~~2028~~2029. Future contractual interest payments (assuming repayments commence on October 1, 2028) associated with the loan are $2019.75 million, with $5.4 million payable within the next 12 months.

Leases

We have entered into lease arrangements for facilities, including corporate, laboratories and office space. As of ~~March~~June 3130, 2026, we had fixed lease payment obligations of $2022.9 million, with $45.62 million payable within the next 12 months. Following the closing of the Lexington Transaction, we assigned our lease for our prior manufacturing facility in Lexington, MA to Genezen. As of ~~March~~June 3130, 2026, we remain obligated under a guarantee of lease payments of $1514.20 million, with the maximum potential exposure under the guarantee decreasing over the remaining lease term through May 2029.

Commitments related to uniQure France SAS acquisition (nominal amounts)

In connection with our acquisition of uniQure France SAS, we entered into commitments to make payments to the former shareholders upon the achievement of certain contractually defined milestones. The commitments include payments related to post-acquisition services that we agreed to as part of the transaction. As of ~~March~~June 3130, 2026, our remaining commitment amounts include EUR 160.0 million ($~~184~~182.03 million) in potential milestone payments associated with Phase III development and the approval of AMT-260 in the U.S. and European Union. The timing of achieving these milestones and consequently the timing of payments, as well as whether the milestones will be achieved at all, is generally uncertain. These payments are owed in euro and have been translated at the foreign exchange rate as of ~~March~~June 3130, 2026 of $1.1514/€1.00. As of ~~March~~June 3130, 2026, we expect these obligations will become payable between 2030 and 2034. If and when due, up to 25% of the milestone payments can be settled with our ordinary shares.

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Commitments related to licensors and financial advisors

We have obligations to make future payments to third parties that become due and payable on the achievement of certain development, regulatory and commercial milestones (such as the start of a clinical trial, filing of a BLA, approval by the FDA or product launch) or as a result of collecting payments related to our sale of the exclusive global rights of HEMGENIX^® to CSL Behring. We also owe payments to a financial advisor related to certain payments we will collect under the CSL Behring Agreement.

The table below summarizes our consolidated cash flow data for the ~~three~~six months ended ~~March~~June 3130, 2026 and 2025:


	~~Three~~Six months ended ~~March~~June 3130,
	2026		2025
	(in thousands)
Cash, cash equivalents and restricted cash at the beginning of the period	$	81,801	$	160,329
Net cash used in operating activities		(3866,~~243~~819)		(4483,~~100~~995)
Net cash generated from investing activities		98151,~~560~~819		2093,~~099~~172
Net cash generated from financing activities		~~550~~249,666		80,~~670~~714
Foreign exchange impact		(1,~~132~~906)		15,~~660~~052
Cash, cash equivalents and restricted cash at the end of period	$	~~141~~414,~~536~~561	$	~~218~~255,~~658~~272

We have previously incurred losses and cumulative negative cash flows from operations since our business was founded by our predecessor entity AMT Therapeutics Holding N.V. in 1998, with the exception of generating income in 2021 after receiving the upfront payment upon closing of the CSL Behring Agreement. We continued to incur losses in the current period. We recorded a net loss of $5381.51 million and $134.6 million in the three and six months ended ~~March~~June 3130, 2026, compared to a net loss of $4337.67 million and $81.4 million during the same period in 2025. As of ~~March~~June 3130, 2026, we had an accumulated deficit of $1,~~382~~463.5 million.

Sources of liquidity

From our first institutional venture capital financing in 2006 through to the current period, we have funded our operations primarily through private and public placements of equity securities, debt securities, pre-funded warrants, and payments from our collaboration partners, as well as $370.1 million through the sale of a portion of royalties due from our collaboration partner CSL Behring in 2023. Between July 2021 and July 2023, we collected $617.4 million from CSL Behring as a result of the sale of HEMGENIX^® to CSL Behring and other milestones collected from CSL Behring, and we are eligible to receive additional milestone payments, as well as royalties (to the extent not owed to settle the liability from the Royalty Financing Agreement) on net sales of HEMGENIX^®.

In June 2026, we received aggregate net proceeds of $242.7 million, after deducting underwriting discounts and commissions and other offering expenses payable by us, following the completion of a follow-on public offering of 5.7 million ordinary shares at a public offering price of $45.50 per ordinary share.

We are subject to certain covenants under the senior secured term loan facility with Hercules and may become subject to covenants under any future indebtedness that could limit our ability to take specific actions, such as incurring additional debt, making capital expenditures, or declaring dividends, which could adversely impact our ability to conduct our business. In addition, our pledge of assets as collateral to secure our obligations under the senior secured term loan facility with Hercules may limit our ability to obtain debt financing.

To the extent we need to finance our cash needs through equity offerings or debt financings, such financing may be subject to unfavorable terms including without limitation, the negotiation and execution of definitive documentation, as well as credit and debt market conditions, and we may not be able to obtain such financing on terms acceptable to us or at all. If financing is not available when needed, including through debt or equity financings, or is available only on unfavorable terms, we may be unable to meet our cash needs. If we raise additional funds through collaborations, strategic alliances or marketing, distribution, or licensing arrangements with third parties, we may have to relinquish valuable rights to our technologies, future revenue streams or product candidates or grant licenses on terms that may not be favorable to us. If we are unable to raise additional funds through equity or debt financings when needed, we may be required to delay, limit, reduce or terminate our product development or future commercialization efforts or grant rights to develop and market product candidates that we would otherwise prefer to develop and market ourselves, which could have a material adverse effect on our business, financial conditions, results of operations and cash flows.

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Net cash used in operating activities

Net cash used in operating activities was $3866.28 million for the ~~three~~six months ended ~~March~~June 3130, 2026 and consisted of a net loss of $53134.56 million, adjusted for non-cash items including depreciation and amortization expense of $37.09 million, amortization of the discount on investment securities of $47.39 million, share-based compensation expense of $510.1 million, $717.24 million of interest expense net of interest paid related the Royalty Financing Agreement, a change in deferred taxes of $02.53 million, changes in the fair value of contingent consideration of $10.31 million, changes in the fair value of the liability related to pre-funded warrants of $312.82 million and unrealized foreign exchange losses of $28.23 million. Net cash used in operating activities also included favorable changes in operating assets and liabilities of $720.10 million. There was a net decrease in accounts receivable, accrued income, prepaid expenses, and other current assets and receivables of $38.8 million. There was a net increase in accounts payable, accrued expenses, ~~other liabilities, income taxes payable~~ and operating leases of $311.31 million.

Net cash used in operating activities was $4484.10 million for the ~~three~~six months ended ~~March~~June 3130, 2025 and consisted of net loss of $4381.64 million adjusted for non-cash items, including depreciation and amortization expense of $48.52 million, amortization of the discount on investment securities of $24.12 million, share-based compensation expense of $49.45 million, $820.46 million of interest expense net of interest paid related to the Royalty Financing Agreement, a change in deferred taxes of $0.59 million, changes in the fair value of contingent consideration of $13.25 million, and unrealized foreign exchange gains of $725.12 million. Net cash used in operating activities also included unfavorable changes in operating assets and liabilities of $612.3 million. There was a net increase in accounts receivable, prepaid expenses, and other current assets and receivables of $36.08 million. There was a net decrease in accounts payable, accrued expenses, other liabilities, and operating leases of $35.36 million.

Net cash generated from investing activities

In the ~~three~~six months ended ~~March~~June 3130, 2026, we generated $98151.68 million from our investing activities compared to $2093.12 million generated during the same period in 2025.


	~~Three~~Six months ended ~~March~~June 3130,
	2026		2025
	(in thousands)
Cash flows from investing activities
Proceeds from maturity of debt securities	$	98384,~~700~~981	$	85213,~~000~~763
Investment in debt securities		—(232,550)		(64120,~~775~~205)
Capital expenditures		(~~140~~612)		(~~126~~386)
Net cash generated from investing activities	$	98151,~~560~~819	$	2093,~~099~~172

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Net cash generated from financing activities

In the ~~three~~six months ended ~~March~~June 3130, 2026, net cash generated from financing activities was $0249.67 million, compared to net cash generated from financing activities of $80.7 million in the same period in 2025.


	~~Three~~Six months ended ~~March~~June 3130,
	2026		2025
	(in thousands)
Cash flows from financing activities
Proceeds from follow-on public offering of ordinary shares, net of issuance costs	$	—242,681	$	80,511
Proceeds from issuance of ordinary shares related to employee stock options and purchase plans		~~550~~6,985		~~159~~203
Net cash generated from financing activities	$	~~550~~249,666	$	80,~~670~~714

Funding requirements

Our future capital requirements will depend on many factors, including but not limited to:

● ~~Investments~~activities to ~~develop~~prepare ~~AMT-130,~~for ~~AMT-191~~the ~~and~~potential commercialization of AMT-~~260~~130 ~~beyond~~for ~~completing~~Huntington’s ~~the ongoing trials~~disease;

● ~~activities~~investments required to ~~prepare~~support ~~for~~ the ~~potential~~MAAs ~~commercialization~~ of AMT-130 ~~for~~or ~~Huntington’s~~generate ~~disease,~~confirmatory ifevidence wein ~~can~~conjunction ~~align on the requirements for marketing authorization~~ with ~~the~~obtaining ~~respective~~accelerated ~~regulatory authorities~~approval;

30● investments in the late stage development of AMT-191 and AMT-260;

~~Table~~● earnout payments we might owe the former shareholders of ~~Contents~~uniQure France SAS, which are subject to achieving specific development and regulatory milestones;

● earnout payments we might owe the former shareholders of uniQure France SAS, which are subject to the achievement of specific development and regulatory milestones;

●

contractual milestone payments and royalties we might be owed in accordance with the CSL Behring Agreement;

●

the scope, timing, results, and costs of our current and planned clinical trials;

●

the scope, obligations and restrictions on our business related to our existing equity, debt or royalty monetization financings and underlying agreements;

●

the extent to which we acquire or in-license other businesses, products, product candidates or technologies;

●

the scope, timing, results and costs of preclinical development and laboratory testing of our additional product candidates;

●

the need for additional resources and related recruitment costs to support the preclinical and clinical development of our product candidates;

●

the need for any additional tests, studies, or trials beyond those originally anticipated to confirm the safety or efficacy of our product candidates and technologies;

●

the cost, timing and outcome of regulatory reviews associated with our product candidates;

●

our ability to enter into collaboration arrangements in the future; and

●

the costs and timing of preparing, filing, expanding, acquiring, licensing, maintaining, enforcing, and prosecuting patents and patent applications, as well as defending any intellectual property-related claims.

Item 3.Quantitative and Qualitative Disclosures about Market Risk

We are exposed to a variety of financial risks in the normal course of our business, including market risk (including currency, price, and interest rate risk), credit risk and liquidity risk. Our overall risk management program focuses on the preservation of capital and the unpredictability of financial markets and has sought to minimize potential adverse effects on our financial performance and position.

Our market risks and exposures to such market risks during the three months ended ~~March~~June 3130, 2026, have not materially changed from our market risks and our exposure to market risk discussed in Part II, Item 7A of our Annual Report.

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Item 4.Controls and Procedures

Evaluation of Disclosure Controls and Procedures

Our management, with the participation of our chief executive officer (“CEO”) and chief financial officer (“CFO”), evaluated the effectiveness of our disclosure controls and procedures (as defined in Rules 13a-15(e) and 15d-15(e) under the Securities Exchange Act of 1934, as amended (the “Exchange Act”) as of ~~March~~June 3130, 2026. Based on such evaluation, our CEO and CFO concluded that as of ~~March~~June 3130, 2026, our disclosure controls and procedures were effective to ensure that information required to be disclosed by it in reports the Company files or submits under the Exchange Act is recorded, processed, summarized and reported within the time periods specified in the rules and forms of the SEC, and that such material information is accumulated and communicated to the Company’s management, including its Principal Executive Officer and Principal Financial Officer, to allow timely decisions regarding required disclosure. Because of the inherent limitations in all control systems, any controls and procedures, no matter how well designed and operated, can provide only reasonable assurance of achieving the desired control objectives, and management necessarily is required to apply its judgment in evaluating the cost-benefit relationship of possible controls and procedures. Furthermore, the Company’s controls and procedures can be circumvented by the individual acts of some persons, by collusion of two or more people or by management override of such control, and misstatements due to error or fraud may occur and not be detected on a timely basis.

Changes in Internal Control over Financial Reporting

During the period covered by this Quarterly Report on Form 10-Q, there were no changes in our internal control over financial reporting (as defined in Rule 13a-15(f) under the Exchange Act) that materially affected, or are reasonably likely to materially affect, our internal control over financial reporting.

During the period covered by this Quarterly Report on Form 10-Q, we implemented a new enterprise resource planning (“ERP”) system in January 2026.

In connection with the ERP implementation, we adapted our internal control activities, including those related to information produced by the entity, user access and segregation of duties, roles and permissions, and approval workflows. The implementation also impacted processes supporting the maintenance of financial records, transaction processing, and financial reporting.

31Part II – OTHER INFORMATION

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Except for the ERP system implementation described above, there were no changes in our internal control over financial reporting (as defined in Rule 13a-15(f) under the Exchange Act) that materially affected, or are reasonably likely to materially affect, our internal control over financial reporting.

Item 1.Legal Proceedings

On February 10, 2026, a class action complaint captioned Christopher Scocco v. uniQure N.V., et al., Case No. 1:26-cv-01124, was filed against us, certain of our executive officers and another party (collectively, “Defendants”) in the United States District Court for the Southern District of New York. The complaint purported to assert claims pursuant to Sections 10(b) and 20(a) of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), and Rule 10b-5 promulgated thereunder, on behalf of a putative class of investors who purchased or otherwise acquired our ordinary shares between September 24, 2025 and October 31, 2025 (the “putative Class Period”). The plaintiff sought to recover damages allegedly caused by purported false and misleading statements and omissions with respect to our Phase I/II study of AMT-130 and the timing of the potential BLA filing for AMT-130.

On April 13, 2026, the court appointed the Oklahoma Firefighters Pension and Retirement System as lead plaintiff (the “Lead Plaintiff”).

On July 15, 2026, the Lead Plaintiff filed an amended complaint. Like the earlier-filed complaint, the amended complaint purports to assert claims pursuant to Sections 10(b) and 20(a) of the Exchange Act and Rule 10b-5, on behalf of a putative class of investors who purchased or otherwise acquired our ordinary shares during the putative Class Period. Lead Plaintiff seeks unspecified damages allegedly caused by purported false and misleading statements and omissions with respect to the import and viability of data from our Phase I/II study of AMT-130 and available pathways to regulatory approval. Defendants’ motion to dismiss the amended complaint is currently due September 14, 2026. Management intends to vigorously defend against the claims in this action.

At each reporting date, we evaluate whether or not a potential loss amount or a potential range of loss is probable and reasonably estimable under the provisions of the authoritative guidance that addresses accounting for contingencies. We have not recorded any liability for any portion of this matter because we believe that liability is not probable and reasonably estimable at this time.

~~Part II – OTHER INFORMATION~~37

~~Item~~Table ~~1.Legal~~of ~~Proceedings~~Contents

Item 1A.Risk Factors

OnFor ~~February 10, 2026,~~ a ~~class~~discussion ~~action~~of ~~complaint~~our ~~captioned Christopher Scocco v. uniQure N.V.~~risks, etplease ~~al.,~~see ~~Case~~“Item No1A. ~~1:26-cv-01124,~~Risk ~~was~~Factors” ~~filed~~in ~~against us, certain of~~ our ~~executive~~Annual ~~officers~~Report ~~and~~on ~~another~~Form ~~party~~10-K ~~in the United States District Court~~ for the ~~Southern~~year ~~District~~ended ofDecember ~~New~~31, ~~York~~2025. The ~~complaint~~information ~~purports~~presented tobelow ~~assert~~updates, ~~claims~~and ~~pursuant~~should tobe ~~Sections~~read ~~10(b)~~in ~~and~~conjunction ~~20(a)~~with, of the ~~Securities~~risk ~~Exchange~~factors ~~Act~~disclosed ofin ~~1934,~~our asAnnual ~~amended,~~Report ~~and~~on ~~Rule~~Form ~~10b~~10-5K ~~promulgated~~for ~~thereunder,~~the onyear ~~behalf~~ended ofDecember ~~a putative class of investors who purchased or otherwise acquired our ordinary shares between September 24, 2025 and October~~ 31, 2025. ~~Plaintiff~~Except ~~seeks~~as topresented ~~recover~~below, ~~damages~~there ~~allegedly~~have ~~caused~~been byno ~~purported~~material ~~false~~changes ~~and~~from ~~misleading~~the ~~statements~~risk ~~and~~factors ~~omissions~~described ~~with~~in ~~respect to~~ our ~~Phase~~Annual ~~I/II~~Report ~~study~~on ofForm ~~AMT~~10-~~130~~K ~~and~~for the ~~timing~~year ofended ~~the~~December ~~potential~~31, ~~BLA filing for AMT-130~~2025. ~~We intend to vigorously defend against the claims in this action.~~

AtWe ~~each~~are ~~reporting~~dependent ~~date,~~on wethe ~~evaluate~~success ~~whether~~of orour ~~not~~lead aclinical ~~potential~~product ~~loss~~candidate, ~~amount~~AMT-130, orfor athe ~~potential~~treatment ~~range~~ of ~~loss~~Huntington’s isdisease. ~~probable~~A ~~and~~failure ~~reasonably estimable under the provisions~~ of ~~the~~AMT-130 ~~authoritative~~in ~~guidance~~clinical ~~that~~development, ~~addresses~~including ~~accounting~~inability ~~for~~to ~~contingencies.~~demonstrate Atsufficient ~~this~~safety ~~time~~or efficacy, weor ~~are~~challenges ~~unable~~associated towith ~~predict~~its ~~the~~regulatory ~~outcome~~approval, ofmanufacturing ~~the class action litigation~~ or ~~reasonably~~commercialization ~~estimate~~could aadversely ~~range~~affect ofour ~~possible losses~~business.

~~Item~~We ~~1A.Risk~~have ~~Factors~~invested a significant portion of our development efforts and financial resources in the development of our lead clinical product candidate, AMT-130 for the treatment of Huntington’s disease.

~~For~~In June 2026, we held a ~~discussion~~Type ofB ~~our~~meeting ~~risks~~with the FDA. Official meeting minutes received in July 2026 confirmed that we and the FDA reached alignment that a BLA submission under the accelerated approval pathway for AMT-130, ~~please~~based ~~see~~on ~~“Item~~the 1Aexisting clinical data, is reasonable. ~~Risk~~In ~~Factors”~~addition, inthe ~~our~~FDA ~~Annual~~seeks ~~Report~~to align on ~~Form~~the 10confirmatory study design prior to the BLA submission, including consideration of a randomized standard-Kof-care control design instead of a sham procedure. The FDA also stated that, in accordance with the FDA’s draft public guidance for accelerated approvals, the ~~fiscal~~confirmatory ~~year~~study ~~ended~~should ~~December~~be 31feasible to conduct within a reasonable timeline and be well underway, ~~2025~~and potentially fully enrolled, at the time of accelerated approval. ~~There~~We ~~have~~expect ~~been~~to submit a BLA in the third quarter of 2026. We can provide no ~~material~~assurance ~~changes~~that towe will align with the ~~risk~~FDA ~~factors~~on ~~disclosed~~a inconfirmatory study design or that our ~~Annual~~BLA ~~Report~~will onbe ~~Form~~accepted 10for review or, if accepted, that our BLA will result in approval of AMT-K130 in Huntington’s disease by the FDA, or that the FDA will agree that we have met the FDA’s requirements for our confirmatory study at the ~~fiscal~~time ~~year~~of ~~ended~~the ~~December~~BLA ~~31,~~action ~~2025~~date.

In March 2026, we held a successful pre-submission meeting with the UK MHRA, and the regulatory submission is progressing as planned for the third quarter of 2026. We can provide no assurance that any such submission will result in approval of AMT-130 in the UK.

There are numerous other factors that could impede or otherwise negatively impact our further development of AMT-130, including, but not limited to: our inability to reach agreement with the FDA on the design of a confirmatory study prior to a BLA submission; potential patient safety issues; our failure to demonstrate sufficient clinical efficacy or durability of response data to warrant further development or approval by the FDA, MHRA or any other regulatory authority; shifting regulatory standards for approval; our current beliefs regarding the further development of and approval pathway for AMT-130, which are based on our interpretation of communications and interactions with regulatory authorities to date, and the success of our efforts to address such communications and interactions; the results from future interim or topline data analyses and readouts from our ongoing Phase I/II trials, including as additional patient data becomes available; any requirement for additional studies to obtain approval, including a confirmatory study; our ability to fund and enroll a confirmatory study, including engaging sites that may be needed to enroll participants in a confirmatory study; the timing, cost and resources associated with our planned marketing applications; our ability to successfully commercialize AMT-130 should we choose to do so without a partner; challenges with potential development or commercial partners, should we choose to pursue further development or commercialization of AMT-130 with a partner; and our ability to fund the further development and commercialization of the AMT-130 program.

32Any one or combination of these factors could force us to halt or discontinue the ongoing clinical trials of AMT-130 or related commercialization efforts or could prevent us from obtaining marketing approval within the predicted timeframes or at all. Certain of these risks are heightened in the context of drug development for rare diseases like Huntington’s disease and novel investigational products like gene therapies in which non-traditional study designs may be utilized to demonstrate efficacy and safety, including open-label studies, single arm studies, studies utilizing active comparators or natural history data, biomarkers or other forms of surrogate endpoints, which may be utilized due to the challenges inherent in designing and conducting clinical trials for severe diseases that progress slowly and that affect small patient populations.

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The FDA has broad discretion with regard to licensure, including through the Accelerated Approval Program, and even if we believe that the Accelerated Approval Program is appropriate for AMT-130, the FDA may require additional studies and trials beyond those that we currently contemplate. Furthermore, even if the FDA reviews a BLA seeking approval, including accelerated approval, there can be no assurance that licensure will be granted on a timely basis, or at all. The FDA may disagree that the design of, or results from, our studies and statistical analysis plan (“SAP”) support accelerated approval. Additionally, the FDA may require us to conduct further studies or trials prior to granting licensure of any type, including by determining that licensure through the Accelerated Approval Program is not appropriate and that our clinical trials and SAP for AMT-130 may not be used to support licensure through the conventional pathway. We might not be able to fulfill the FDA’s requirements in a timely manner, which would cause delays, or licensure might not be granted because our submission is deemed incomplete by the FDA. Furthermore, the feedback or requests we receive from the FDA, or other non-U.S. regulatory authority, may be difficult or impossible to implement. There also can be no assurance that after subsequent FDA feedback we will continue to pursue licensure, including through the Accelerated Approval Program or otherwise. A failure to obtain licensure for AMT-130 could delay or prevent our ability to launch and commercialize AMT-130 in the U.S., could result in a longer time period to pursue licensure of our other clinical product candidates or increase the cost of development of our other clinical product candidates, and could significantly harm our financial position and competitive position in the marketplace.

We cannot be certain that AMT-130, or any of our clinical product candidates, will be successful in clinical trials or ultimately receive regulatory approval. If we were required, or if we chose, to discontinue development of AMT-130 or any other current or future product candidate, or if any of them were to fail to receive regulatory approval or achieve sufficient market acceptance, we could be prevented from or significantly delayed in achieving profitability and our business would be adversely affected.

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Item 2.Unregistered Sales of Equity Securities, Use of Proceeds, and Issuer Purchases of Equity Securities

None.

Item 3.Defaults Upon Senior Securities

None.

Item 4.Mine Safety Disclosures

Not applicable.

Item 5.Other Information

~~During~~Entry ~~the~~into ~~three months ended March 31, 2026, the following adopted~~ a ~~“Rule~~Material ~~10b5-1~~Definitive ~~trading arrangement” (as defined in Item 408(a) of Regulation S-K) that is intended to satisfy the affirmative defense of Rule 10b5-1(c) under the Exchange Act and our policies on insider trading:~~Agreement


Name & Title	Date Adopted(1)	Aggregate Number of Ordinary Shares to be Purchased or Sold Pursuant to Trading Arrangement	Expiration Date(2)
Madhavan Balachandran, Non-executive Director	March 10, 2026	21,685	December 31, 2026

On July 27, 2026, we and certain of our affiliates entered into Amendment No. 4 to Third Amended and Restated Loan and Security Agreement (the “Amendment”) with Hercules, which amended certain terms of the Third Amended and Restated Loan and Security Agreement, dated December 15, 2021 by and among us, certain of our subsidiaries, and Hercules, as amended by that certain Amendment No. 1 to Third Amended and Restated Loan and Security Agreement, dated as of May 12, 2023, Consent and Amendment No. 2 to Third Amended and Restated Loan and Security Agreement, dated as of June 28, 2024, and Amendment No. 3 to Third Amended and Restated Loan and Security Agreement.

The Amendment, among other things, extends the period we can draw the $100.0 million term loan tranche from June 15, 2027 to September 30, 2027. Except as provided for in the amendment, the terms of borrowing under the 2026 Amended Facility otherwise remain unchanged.

The foregoing description of the Amendment does not purport to be complete and is qualified in its entirety by reference to the full text of the Amendment, a copy of which is filed as Exhibit 10.2 hereto and is incorporated herein by reference.

Adoption of 10b5-1 Trading Plans by Our Officers and Directors

During the three months ended June 30, 2026, the following adopted a “Rule 10b5-1 trading arrangement” (as defined in Item 408(a) of Regulation S-K) that is intended to satisfy the affirmative defense of Rule 10b5-1(c) under the Exchange Act and our policies on insider trading:


Name & Title	Date Adopted⁽¹⁾	Aggregate Number of Ordinary Shares to be Purchased or Sold Pursuant to Trading Arrangement	Expiration Date⁽²⁾
David Meek, Non-executive Director	May 26, 2026	25,860	December 18, 2026

⁽¹⁾ Date of adoption of this Rule 10b5-1 trading arrangement is in accordance with applicable SEC rules and regulations. The first trade pursuant to this Rule 10b5-1 trading arrangement will be, in accordance with applicable SEC rules and regulations, on a date after the date of adoption of the Rule 10b5-1 trading arrangement, to the extent triggered under its terms.

⁽²⁾The Rule 10b5-1 trading arrangement is scheduled to expire on the date listed in the table, subject to earlier termination upon the sale of all shares subject to the Rule 10b5-1 trading arrangement, or as otherwise provided in the Rule 10b5-1 trading arrangement.

Other than those disclosed above, none of our directors or officers adopted, modified, or terminated a “Rule 10b5-1 trading arrangement” or “non-Rule 10b5-1 trading arrangement,” in each case as defined in Item 408 of Regulation S-K, during the three months ended ~~March~~June 3130, 2026.

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Item 6.Exhibits

See the Exhibit Index immediately preceding the signature page to this Quarterly Report on Form 10-Q for a list of exhibits filed or furnished with this report, which Exhibit Index is incorporated herein by reference.

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EXHIBIT INDEX



10.1*+	Termination and Amendment Agreement, dated April 15, 2026, by and between uniQure biopharma B.V. and CSL Behring LLC.
3.1	Amended Articles of Association of uniQure N.V. (incorporated by reference to Exhibit 3.1 of the Company’s Current Report on Form 8-K (file no. 001-36294) filed with the SEC on June 15, 2026).

10.1	Amended and Restated 2014 Share Incentive Plan of uniQure N.V. (incorporated by reference to Exhibit 10.1 of the Company’s Current Report on Form 8-K (file no. 001-36294) filed with the SEC on June 15, 2026).

10.2*+	Amendment No. 4 to Third Amended and Restated Loan and Security Agreement, dated July 27, 2026, by and among uniQure biopharma, B.V., uniQure, Inc., uniQure IP B.V., uniQure N.V. and Hercules Capital, Inc.

31.1*	Certification of Chief Executive Officer pursuant to Rule 13a-14(a) or 15d-14(a) of the Securities and Exchange Act of 1934, as amended, as adopted pursuant to Section 302 of the Sarbanes-Oxley Act of 2002.

31.2*	Certification of Chief Financial Officer pursuant to Rule 13a-14(a) or 15d-14(a) of the Securities and Exchange Act of 1934, as amended, as adopted pursuant to Section 302 of the Sarbanes-Oxley Act of 2002.

32.1±	Certification of Principal Executive Officer and Principal Financial Officer pursuant to 18 U.S.C. Section 1350, as adopted pursuant to Section 906 of the Sarbanes-Oxley Act of 2002.

101*	The following financial information from our Quarterly Report on Form 10-Q for the period ended ~~March~~June 3130, 2026, filed with the Securities and Exchange Commission on ~~May~~July 529, 2026, is formatted in Inline Extensible Business Reporting Language (“iXBRL”): (i) Consolidated Balance Sheets; (ii) Consolidated Statements of Operations and Comprehensive Loss; (iii) Consolidated Statements of Shareholders’ Equity; (iv) Consolidated Statements of Cash Flows; and (v) Notes to Consolidated Financial Statements (tagged as blocks of text).

104*	The cover page from our Quarterly Report on Form 10-Q for the period ended ~~March~~June 3130, 2026, filed with the Securities and Exchange Commission on ~~May~~July 529, 2026, is formatted in Inline Extensible Business Reporting Language (“iXBRL”).

* Filed herewith.

± Furnished herewith.

+Portions of this exhibit have been omitted pursuant to Item 601(b)(10)(iv) of Regulation S-K.

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SIGNATURE

Pursuant to the requirements of the Securities Exchange Act of 1934, as amended, the registrant has duly caused this report to be signed on its behalf by the undersigned thereunto duly authorized.


	UNIQURE N.V.


	By: /s/ Matthew Kapusta Matthew Kapusta Chief Executive Officer (Principal Executive Officer)


	By: /s/ Christian Klemt Christian Klemt Chief Financial Officer (Principal Financial and Accounting Officer)

Date: ~~May~~July 529, 2026

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