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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 September 30, 2025

	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

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 November 6, 2025, the registrant had 62,291,663 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	22

Item 3	Quantitative and Qualitative Disclosures About Market Risk	42

Item 4	Controls and Procedures	42

	PART II – OTHER INFORMATION

Item 1	Legal Proceedings	42

Item 1A	Risk Factors	43

Item 2	Unregistered Sales of Equity Securities, Use of Proceeds, and Issuer Purchases of Equity Securities	89

Item 3	Defaults Upon Senior Securities	89

Item 4	Mine Safety Disclosures	89

Item 5	Other Information	89

Item 6	Exhibits	89

Table of Contents

SPECIAL CAUTIONARY NOTICE REGARDING FORWARD-LOOKING STATEMENTS

This Quarterly Report on Form 10-Q contains “forward-looking statements” as defined under 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, include, but are not limited to, statements concerning: our financial position, revenues, costs, expenses, 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 or completion of or availability of results from any preclinical studies and clinical trials or for the submission, review or approval of any regulatory filing, including the timing of the Company’s Biologics License Application for AMT-130 to the United States Food and Drug Administration (the “FDA”); the anticipated or ultimate outcome, and timing, of interactions with regulatory agencies, including the FDA; the timing of, and our ability to, obtain and maintain regulatory approvals for any of our product candidates; the potential benefits that may be derived from any 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 the planning for potential commercialization of AMT-130; our ability to achieve long-term growth; our financial performance; developments and projections relating to our competitors in the industry; and our liquidity and working capital requirements.

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 February 27, 2025 (the “Annual Report”), our Quarterly Reports on Form 10-Q filed with the SEC on May 9, 2025 and July 29, 2025, 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


	September 30,		December 31,
	2025		2024

	(in thousands, except share and per share amounts)
Current assets
Cash and cash equivalents	$	597,056	$	158,930
Current investment securities		97,189		208,591
Accounts receivable		3,883		5,881
Prepaid expenses		10,326		9,281
Other current assets and receivables		7,739		7,606
Total current assets		716,193		390,289
Non-current assets
Property, plant and equipment, net of accumulated depreciation of $43.5 million as of September 30, 2025 and $31.7 million as of December 31, 2024		15,402		20,424
Other investments		29,972		27,464
Operating lease right-of-use assets		13,079		13,647
Intangible assets, net, including in-process research and development assets of $62.9 million as of September 30, 2025 and $55.6 million as of December 31, 2024		74,144		71,043
Goodwill		25,327		22,414
Deferred tax assets, net		8,772		9,856
Other non-current assets		5,493		1,399
Total non-current assets		172,189		166,247
Total assets	$	888,382	$	556,536
Current liabilities
Accounts payable	$	6,228	$	7,227
Accrued expenses and other current liabilities		51,947		28,932
Liability related to pre-funded warrants		30,722		—
Income taxes payable		7,821		293
Current portion of operating lease liabilities		3,914		3,601
Total current liabilities		100,632		40,053
Non-current liabilities
Long-term debt		51,880		51,324
Liability from royalty financing agreement		465,507		434,930
Operating lease liabilities, net of current portion		10,361		11,136
Contingent consideration		17,754		10,860
Deferred tax liability, net		7,958		7,043
Other non-current liabilities, net of current portion		5,544		7,942
Total non-current liabilities		559,004		523,235
Total liabilities		659,636		563,288
Commitments and contingencies
Shareholders' equity / (deficit)
Ordinary shares, €0.05 par value: 80,000,000 shares authorized as of September 30, 2025 and December 31, 2024 and 62,165,900 and 48,988,087 ordinary shares issued and outstanding as of September 30, 2025 and December 31, 2024, respectively		3,678		2,945
Additional paid-in-capital		1,575,663		1,173,068
Accumulated other comprehensive loss		(58,745)		(52,800)
Accumulated deficit		(1,291,850)		(1,129,965)
Total shareholders' equity / (deficit)		228,746		(6,752)
Total liabilities and shareholders' equity / (deficit)	$	888,382	$	556,536

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 September 30,				Nine months ended September 30,
	2025		2024		2025		2024

	(in thousands, except share and per share amounts)				(in thousands, except share and per share amounts)
License revenues	$	3,582	$	2,111	$	10,411	$	5,182
Contract manufacturing revenues		—		—		—		6,114
Collaboration revenues		119		176		119		10,602
Total revenues		3,701		2,287		10,530		21,898
Operating expenses:
Cost of license revenues		(398)		(264)		(1,251)		(648)
Cost of contract manufacturing revenues		—		(757)		—		(17,060)
Research and development expenses		(34,366)		(30,595)		(105,889)		(104,942)
Selling, general and administrative expenses		(19,438)		(11,575)		(43,846)		(41,279)
Total operating expenses		(54,202)		(43,191)		(150,986)		(163,929)
Other income		1,510		2,591		12,413		5,950
Other expense		(2,044)		(1,915)		(6,188)		(2,385)
Loss from operations		(51,035)		(40,228)		(134,231)		(138,466)
Interest income		3,503		4,866		11,154		17,179
Interest expense		(16,721)		(16,611)		(47,421)		(48,865)
Foreign currency (losses) / gains, net		(428)		7,564		25,382		5,430
Other non-operating losses, net		(7,222)		—		(7,222)		—
Loss before income tax expense	$	(71,903)	$	(44,409)	$	(152,338)	$	(164,722)
Income tax (expense) / benefit		(8,626)		31		(9,547)		(1,573)
Net loss	$	(80,529)	$	(44,378)	$	(161,885)	$	(166,295)
Other comprehensive loss:
Foreign currency translation gains / (losses), net		158		(415)		(5,945)		(3,252)
Defined benefit pension gain, net of taxes		—		37		—		107
Total comprehensive loss	$	(80,371)	$	(44,756)	$	(167,830)	$	(169,440)
Loss per ordinary share - basic and diluted
Basic and diluted net loss per ordinary share	$	(1.38)	$	(0.91)	$	(2.90)	$	(3.42)
Weighted average shares - basic and diluted		58,516,415		48,718,533		55,880,318		48,576,339

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’ EQUITY / (DEFICIT)
FOR THE THREE MONTHS ENDED SEPTEMBER 30, 2025 AND 2024


						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 June 30, 2024	48,694,569	$	2,930	$	1,162,823	$	(56,320)	$	(1,012,326)	$	97,107
Loss for the period	—		—		—		—		(44,378)		(44,378)
Other comprehensive loss, net	—		—		—		(378)		—		(378)
Exercise of share options	3,900		—		32		—		—		32
Restricted share units distributed during the period	40,405		2		(2)		—		—		—
Share-based compensation expense	—		—		3,438		—		—		3,438
Balance at September 30, 2024	48,738,874	$	2,932	$	1,166,291	$	(56,698)	$	(1,056,704)	$	55,821

Balance at June 30, 2025	54,865,673	$	3,250	$	1,262,971	$	(58,903)	$	(1,211,321)	$	(4,003)
Loss for the period	—		—		—		—		(80,529)		(80,529)
Other comprehensive gain, net	—		—		—		158		—		158
Follow-on public offering	6,736,841		394		299,832		—		—		300,226
Tax benefit related to historical share issuance costs	—		—		862						862
Exercises of share options	532,316		32		8,478		—		—		8,510
Restricted share units distributed during the period	31,070		2		(2)		—		—		—
Share-based compensation expense	—		—		3,522		—		—		3,522
Balance at September 30, 2025	62,165,900	$	3,678	$	1,575,663	$	(58,745)	$	(1,291,850)	$	228,746

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’ EQUITY / (DEFICIT)
FOR THE NINE MONTHS ENDED SEPTEMBER 30, 2025 AND 2024


						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, 2023	47,833,830	$	2,883	$	1,148,749	$	(53,553)	$	(890,409)	$	207,670
Loss for the period	—		—		—		—		(166,295)		(166,295)
Other comprehensive loss, net	—		—		—		(3,145)		—		(3,145)
Exercises of share options	3,900		—		32		—		—		32
Restricted share units distributed during the period	890,994		48		(48)		—		—		—
Share-based compensation expense	—		—		17,509		—		—		17,509
Issuance of ordinary shares relating to employee stock purchase plan	10,150		1		49		—		—		50
Balance at September 30, 2024	48,738,874	$	2,932	$	1,166,291	$	(56,698)	$	(1,056,704)	$	55,821

Balance at December 31, 2024	48,988,087	$	2,945	$	1,173,068	$	(52,800)	$	(1,129,965)	$	(6,752)
Loss for the period	—		—		—		—		(161,885)		(161,885)
Other comprehensive loss, net	—		—		—		(5,945)		—		(5,945)
Follow-on public offerings	11,810,370		655		380,082		—		—		380,737
Tax benefit related to historical share issuance costs	—		—		862						862
Exercises of share options	553,556		33		8,680		—		—		8,713
Restricted and performance share units distributed during the period	813,887		45		(45)		—		—		—
Share-based compensation expense	—		—		13,016		—		—		13,016
Balance at September 30, 2025	62,165,900	$	3,678	$	1,575,663	$	(58,745)	$	(1,291,850)	$	228,746

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


	Nine months ended September 30,
	2025		2024

	(in thousands)
Cash flows from operating activities
Net loss	$	(161,885)	$	(166,295)
Adjustments to reconcile net loss to net cash used in operating activities:
Depreciation and amortization		11,789		8,285
Amortization of discount on investment securities		(6,091)		(8,472)
Share-based compensation expense		13,016		17,509
Royalty financing agreement interest expense, net of interest paid		29,209		33,107
Deferred tax expense		1,979		1,573
Changes in fair value of contingent consideration		5,293		(2,032)
Changes in fair value of liability related to pre-funded warrants		5,722		—
Unrealized foreign exchange gains, net		(24,534)		(5,726)
Allowance for inventory write-downs		—		6,297
Other items, net		(3,013)		(2,460)
Changes in operating assets and liabilities:
Accounts receivable, prepaid expenses, and other current assets and receivables		2,880		(5,764)
Inventories		—		(3,876)
Accounts payable		(1,648)		(512)
Income tax payable		7,568		167
Accrued expenses, other liabilities, and operating leases		19,321		(1,685)
Net cash used in operating activities		(100,394)		(129,884)
Cash flows from investing activities
Proceeds on maturity of debt securities		337,195		480,806
Investment in debt securities		(217,124)		(279,586)
Divestment of commercial manufacturing facility		—		(8,300)
Purchases of property, plant, and equipment		(320)		(3,328)
Net cash generated from investing activities		119,751		189,592
Cash flows generated from / (used in) financing activities
Proceeds from follow-on public offerings of ordinary shares, net of issuance costs		380,737		—
Proceeds from issuance of pre-funded warrants, net of issuance costs		23,499		—
Proceeds from issuance of ordinary shares related to employee stock options and purchase plans		8,713		81
Repayment of long-term debt		—		(53,050)
Net cash generated from / (used in) financing activities		412,949		(52,969)
Currency effect on cash, cash equivalents and restricted cash		5,914		1,796
Net increase in cash, cash equivalents and restricted cash		438,220		8,535
Cash, cash equivalents and restricted cash at beginning of period		160,329		244,544
Cash, cash equivalents and restricted cash at the end of period	$	598,549	$	253,079
Cash and cash equivalents	$	597,056	$	251,626
Restricted cash related to leasehold and other deposits		1,493		1,453
Total cash, cash equivalents and restricted cash	$	598,549	$	253,079
Supplemental cash flow disclosures:
Cash paid for interest	$	(16,659)	$	(16,892)
Non-cash decrease in accounts payables and accrued expenses and other current liabilities related to purchases of property, plant, and equipment	$	(54)	$	(673)

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, 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, 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 nine months ended September 30, 2025, are not necessarily indicative of the results to be expected for the full year ending December 31, 2025, 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, 2024 filed by the Company with the SEC on February 27, 2025 (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, disclosure of contingent liabilities at the date of the financial statements and 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, 2024, 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 nine months ended September 30, 2025, except as noted below:

Liability related to pre-funded warrants

As part of the Company’s September 2025 follow-on public offering, the Company issued pre-funded warrants (the “Pre-Funded Warrants”), denominated in U.S. dollars. As the Pre-Funded Warrants are not denominated in the Company’s functional currency, they are not indexed to the Company’s ordinary shares in accordance with ASC 815-40, Derivatives and Hedging, Contracts in Entity’s Own Equity. The obligation to issue ordinary shares at an offering price of $47.50 per ordinary share, less a $0.0001 per ordinary share exercise price, is classified as a liability. The liability related to pre-funded warrants is accounted for at fair value in accordance with ASC 820, Fair Value Measurement. Changes in the fair value of the liability related to pre-funded warrants are recognized within Other non-operating items, net in the Company’s Consolidated Statements of Operations and Comprehensive Loss. The liability related to pre-funded warrants is classified as current due to the Pre-Funded Warrant holder’s right to exercise at any time. The Company made an accounting policy election to exclude the Pre-Funded Warrants from the denominator in the computation of basic net loss per ordinary share, as such instruments are not considered outstanding ordinary shares.

2.5Recent accounting pronouncements

There have been no new accounting pronouncements or changes to accounting pronouncements during the nine months ended September 30, 2025, 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	Other investments

Convertible promissory note

The Company has an investment in a convertible promissory note with a nominal amount of $12.5 million, bearing interest at 8.0% per annum. The convertible promissory note was issued in July 2024 and matures in October, 2029. The convertible promissory note had a balance of $14.4 million as of September 30, 2025 (December 31, 2024: $13.7 million) and is presented within Other investments on the Company’s Consolidated Balance Sheets. In the three and nine months ended September 30, 2025, the Company recognized $0.2 million and $0.7 million of interest income, respectively, compared to nil during the same periods in 2024. As of September 30, 2025, the Company has not recorded an allowance for credit losses related to the convertible promissory note.

Investments valued under measurement alternative

The Company has investments in non-publicly traded equity securities without readily determinable fair values. These investments are accounted for at cost, less impairment and adjusted to fair value if there are observable price changes in orderly transactions for identical or similar investments using the measurement alternative in accordance with ASC 321, Investments – Equity Securities. The carrying value of these investments as of September 30, 2025 and December 31, 2024 was $15.6 million and $13.8 million, respectively, as presented within Other Investments on the Company’s Consolidated Balance Sheets. The Company did not recognize any gains, losses, or impairments relating to investments carried under the measurement alternative in the three and nine month periods ended September 30, 2025 and September 30, 2024.

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4	Investment securities

The following tables summarize the Company’s investments in government debt securities as of September 30, 2025 and December 31, 2024:


	At September 30, 2025
	Amortized cost		Gross unrealized holding gains		Gross unrealized holding losses		Estimated fair value

	(in thousands)
Current investments:
Government debt securities (held-to-maturity)	$	97,189	$	51	$	—	$	97,240
Total	$	97,189	$	51	$	—	$	97,240

	At December 31, 2024
	Amortized cost		Gross unrealized holding gains		Gross unrealized holding losses		Estimated fair value

	(in thousands)
Current investments:
Government debt securities (held-to-maturity)	$	208,591	$	164	$	—	$	208,755
Total	$	208,591	$	164	$	—	$	208,755

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 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 (September 30, 2025: $119.3 million, December 31, 2024: $15.3 million).

Inputs to the fair value of the investments are considered Level 2 inputs.

5	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 Company’s Consolidated Balance Sheets approximate their fair values due to their short-term maturities.

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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 September 30, 2025 and December 31, 2024:


	Quoted prices in active markets (Level 1)		Significant other observable inputs (Level 2)		Significant unobservable inputs (Level 3)		Total		Classification in Consolidated balance sheets

		(in thousands)
Balance at December 31, 2024
Assets:
Cash and cash equivalents	$	158,930	$	—	$	—	$	158,930	Cash and cash equivalents
Restricted cash		1,399		—		—		1,399	Other non-current assets
Total assets	$	160,329	$	—	$	—	$	160,329
Liabilities:
Contingent consideration		—		—		10,860		10,860	Contingent consideration
Consideration for post-acquisition services		—		—		370		370	Other non-current liabilities
Total liabilities	$	—	$	—	$	11,230	$	11,230
Balance at September 30, 2025
Assets:
Cash and cash equivalents	$	597,056	$	—	$	—	$	597,056	Cash and cash equivalents
Restricted cash		1,493		—		—		1,493	Other non-current assets
Total assets	$	598,549	$	—	$	—	$	598,549
Liabilities:
Liability related to pre-funded warrants		—		30,722		—		30,722	Liability related to pre-funded warrants
Contingent consideration		—		—		17,754		17,754	Contingent consideration
Consideration for post-acquisition services		—		—		605		605	Other non-current liabilities
Total liabilities	$	—	$	30,722	$	18,359	$	49,081

Contingent consideration

The Company is required to pay up to EUR 143.1 million (or $167.9 million based on the foreign exchange rate on September 30, 2025) 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 September 30, 2025 was $17.8 million (December 31, 2024: $10.9 million) using discount rates of approximately 12.1% to 12.5% (December 31, 2024: 15.3% to 16.2%).

If, as of September 30, 2025, 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 $57.2 million. If, as of September 30, 2025, 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 between December 31, 2024 and September 30, 2025:


	Amount of
	contingent
	consideration
	2025
	(in thousands)
Balance at December 31, 2024	$	10,860
Unrealized change in fair value (presented within research and development expenses)		5,293
Currency translation effects		1,601
Balance at September 30, 2025	$	17,754

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The Company classified the total contingent consideration liability as non-current as of September 30, 2025 and December 31, 2024. The classification of the contingent consideration within the Company’s 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.

Investment securities

Refer to Note 4 “Investment securities” for the fair value of the investment securities as of September 30, 2025 and December 31, 2024.

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. 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 liability related to pre-funded warrants is accounted for at fair value in accordance with ASC 820, Fair Value Measurement, with the total gross proceeds received with respect to the Pre-Funded Warrants determined as reflective of the fair value of the obligation on the date of issuance. Changes in fair value of the liability related to pre-funded warrants are recognized within Other non-operating losses, net in the Company’s Consolidated Statements of Operations and Comprehensive Loss.

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
	2025
	(in thousands)
Balance at December 31, 2024	$	—
Issuance of pre-funded warrants in connection with follow-on public offering		25,000
Unrealized change in fair value		5,722
Balance at September 30, 2025	$	30,722

The fair value of the liability related to pre-funded warrants as of September 30, 2025 was $30.7 million (December 31, 2024: nil), classified as current. Classification is based upon the Pre-Funded Warrant holder’s right to exercise at any time. During the three and nine months ended September 30, 2025, the Company recorded a $5.7 million remeasurement in the fair value of the liability related to pre-funded warrants, respectively.

6	Accrued expenses and other current liabilities

Accrued expenses and other current liabilities include the following items:


	September 30,		December 31,
	2025		2024

	(in thousands)
Personnel-related accruals and liabilities (As of September 30, 2025, $15.7 million (December 31, 2024: $0.1 million) related to cash proceeds from employees stock option exercises)	$	26,526	$	12,290
Accruals for goods received from and services provided by vendors-not yet billed		16,941		10,109
Current portion of firm purchase commitment liability		3,773		1,582
Liability owed to the Purchaser pursuant to the Royalty Financing Agreement (refer to Note 8 "Royalty Financing Agreement")		3,583		4,951
Other current liabilities		1,124		—
Total	$	51,947	$	28,932

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7Long-term debt

On June 14, 2013, the Company entered into a venture debt loan facility with Hercules Capital, Inc. (“Hercules”). The facility has been amended at various times, including on July 22, 2024 (the “2024 Amended Facility”) and most recently on September 22, 2025 (the “2025 Amended Facility), upon which date the amendment closed (the “Amendment Closing Date”).

On the Amendment Closing Date, the Company drew down $50.0 million (the “Tranche 1 Advance”) of the 2025 Amended Facility to repay the $50.0 million then outstanding under the 2024 Amended Facility. A facility charge of $0.2 million was incurred on the Amendment Closing Date. The 2024 Amended Facility was due on January 7, 2027 and bore interest at a rate equal to the greater of (i) 7.95% and (ii) 7.95% plus the prime rate less 3.25% per annum, reflecting a floating rate of 11.95% at the Amendment Closing Date.

Pursuant to the 2025 Amended Facility, additional term loans of $125.0 million were made available. The Company may draw down (i) an additional $100.0 million in two increments of at least $25.0 million (the “Tranche 2 Advances”), following the approval of a Biologics License Application (“BLA”) for AMT-130 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, and (ii) an additional $25.0 million in a single advance (the “Tranche 3 Advance”), subject to approval by Hercules. Advances under the 2025 Amended Facility bear interest at a rate equal to the greater of (i) 9.45% and (ii) the sum of the prime rate and 2.45%, reflecting a current floating rate of 9.70%.

The 2025 Amended Facility matures on October 1, 2030, upon which date the Company is required to repay the principal balance and any unpaid interest on advances received. The interest-only period of the 2025 Amended Facility is effective through October 1, 2028. The interest-only period will be extended to October 1, 2029 if the BLA for AMT-130 is approved prior to June 15, 2027. The 2025 Amended Facility will be interest-only if certain commercial milestones are met prior to December 31, 2028.

The Company may prepay all or part of the outstanding principal together with a prepayment charge of 1.50% if repayment occurs within the first 30 months following the Amendment Closing Date, and thereafter, with a prepayment charge of 0.75%. Pursuant to the terms of the 2025 Amended Facility, the Company owes a back-end fee on December 1, 2025. Absent a default or prepayment of the loan, a back-end fee of $0.6 million is due on January 5, 2027. An end-of-term charge of 2.75% of the aggregate principal outstanding is due if repaid within 18 months of the Amendment Closing Date, otherwise 5.50% if repaid thereafter.

The total principal outstanding as of September 30, 2025 under 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 $52.4 million as of September 30, 2025, compared to $51.9 million as of December 31, 2024, and is recorded net of discount and debt issuance costs. The foreign currency gain on the loan facilities in the three and nine months ended September 30, 2025 was $2.8 million and $9.2 million, respectively, compared to a foreign currency gain of $2.6 million and a foreign currency loss of $0.4 million, respectively, during the same periods in 2024.

Interest expense during the three and nine months ended September 30, 2025 was $1.8 million and $5.4 million, respectively, compared to $3.7 million and $11.1 million, respectively, during the same periods in 2024. The Company repaid $50.0 million of the outstanding principal amount of the 2024 Amended Facility on July 19, 2024.

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Under the 2025 Amended Facility, the Company must remain current in its periodic reporting requirements. Unless the Company maintains a minimum market capitalization threshold of at least $1,200.0 million, the Company, beginning January 1, 2027 (the “Test Date”), is required at all times to keep a minimum cash balance deposited in bank accounts in the U.S. of at least the lesser of (i) 65% of the outstanding balance of aggregate principal due, provided that such percentage will be reduced to 50% upon the occurrence of the approval of a BLA for AMT-130 prior to June 15, 2027, and further reduced to 35% upon the occurrence of the contractually defined commercial milestones or (ii) 100% of worldwide cash and cash equivalents. The 2025 Amended Facility also provides for a certain performance covenant related to AMT-130 net product revenue which would apply nine months past a potential BLA approval of AMT-130, unless the Company maintains a minimum market capitalization threshold of at least $1,200.0 million. The 2025 Amended Facility restricts the Company’s ability, among other things, to incur future indebtedness and obtain additional debt financing, make investments in securities or in other companies, transfer assets, perform certain corporate changes, own or hold certain digital assets, make loans to employees, officers, and directors, and make dividend payments and other distributions to its shareholders. The 2025 Amended Facility is secured by a direct or indirect pledge of the Company’s total assets of $888.4 million, less $80.6 million of cash and cash equivalents and other current assets held by the Company, and $88.7 million of other current assets and investments held by uniQure France SAS as well as receivables sold to HemB SPV, L.P. as described in Note 8 “Royalty Financing Agreement”. In accordance with the 2025 Amended Facility, the Company was in compliance with all applicable covenants as of September 30, 2025.

8Royalty Financing Agreement

In May 2023, the Company entered into a royalty purchase agreement (the “Royalty Financing Agreement”) with HemB SPV, L.P. (the “Purchaser”). Under the terms of the Royalty Financing Agreement, the Company received an upfront payment of $375.0 million in exchange for its rights to the lowest royalty tier on CSL Behring LLC’s (“CSL Behring”) worldwide net sales of etranacogene dezaparvovec-drlb (HEMGENIX®) for certain current and future royalties due to the Company. The Company will be obligated to pay $25.0 million of the first worldwide sales milestone payment from CSL Behring, if received, to the Purchaser.

The Purchaser will receive 1.85 times the upfront payment (or $693.8 million) until June 30, 2032 (“First Hard Cap Date”) if such thresholds are met or, if such cap is not met by June 30, 2032, up to 2.25 times the upfront payment through December 31, 2038 (“Second Hard Cap Date”). If, on or prior to the defined dates for each cap amount, the total amount of royalty payments received by the Purchaser equals or exceeds the cap amount applicable to such date, the Royalty Financing Agreement will automatically terminate and all rights to the HEMGENIX® royalty payments will revert back to the Company. The Company has no obligation to repay any amounts received from the Purchaser in the event that the applicable cap amount is not reached during the term of the Royalty Financing Agreement.

The Company has retained the rights to all other royalties, as well as contractual milestones totaling up to $1.3 billion, under the terms of the commercialization and license agreement with CSL Behring (the “CSL Behring Agreement”).

Net proceeds from the Royalty Financing Agreement were $370.1 million. The Company initially recorded these net proceeds as Liability from royalty financing agreement at their fair market value on its Consolidated Balance Sheet as of the date the transaction closed on June 5, 2023. Following the initial recognition, the Company records the debt at amortized cost.

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As of September 30, 2025 the Company expects to satisfy its commitment to the Purchaser prior to the Second Hard Cap Date. The Company records the difference of $473.7 million between the total expected payments of $843.8 million to the Purchaser and the $370.1 million net proceeds as interest expense over the term using the effective interest rate method. The Company determined the effective interest rate based on the projected cash flows up to the Second Hard Cap Date. As of September 30, 2025 the effective interest rate is expected to be within a range of 10.5% to 12.0% per annum, compared to a range of 12.0% to 13.5% per annum as of September 30, 2024. The Company would have recorded the following amounts of interest expense if it had used the effective interest rate at the lower and upper end of this range:


	Three months ended September 30,				Nine months ended September 30,
	2025		2024		2025		2024

		(in millions)				(in millions)
Interest expense at effective interest rate of 10.5% per annum for the three months and nine months ended September 30, 2025, and 12.0% per annum for the three and nine months ended September 30, 2024, respectively	$	12.3	$	12.9	$	35.8	$	37.4
Interest recorded in the Consolidated Statements of Operations and Comprehensive Loss		13.8		12.8		40.9		37.7
Interest expense at effective interest rate of 12.0% per annum for the three months and nine months ended September 30, 2025, and 13.5% per annum for the three and nine months ended September 30, 2024, respectively		14.2		14.6		41.1		42.3

The Company prospectively updates the effective interest rate at each reporting date based on updated cash flow projections.

The following table presents the movement in the liability related to the Royalty Financing Agreement between December 31, 2024 and September 30, 2025:


	Amount of liability
	(in thousands)
Balance as of December 31, 2024 (includes $5.0 million presented as "Accrued expenses and other current liabilities")	$	439,881
Royalty payments to the Purchaser		(11,675)
Interest expense for the period		40,884
Balance as of September 30, 2025 (includes $3.6 million presented as "Accrued expenses and other current liabilities")	$	469,090

9Shareholders' equity / (deficit)

In January 2025, the Company received net proceeds of $70.1 million, after deducting underwriting discounts and commissions and other offering expenses payable by the Company, through a follow-on public offering of 4.4 million ordinary shares at a public offering price of $17.00 per ordinary share.

In February 2025, the Company received an additional $10.4 million in net proceeds after related offering expenses upon the underwriters’ exercise of their option to purchase an additional 0.7 million ordinary shares at the public offering price.

In July 2025, the Company entered into a Sales Agreement with Leerink Partners LLC (“Leerink Partners”) with respect to an at-the-market (“ATM”) offering program, under which the Company may, from time to time and at the Company’s sole discretion, offer and sell through Leerink Partners, acting as agent, the Company’s ordinary shares, up to an aggregate offering of $200.0 million. As of September 30, 2025, no sales have been made under the ATM offering program.

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In September 2025, the Company received net proceeds of $323.7 million, after deducting underwriting discounts and commissions and other offering expenses payable by the Company, through a follow-on public offering of 6.7 million ordinary shares, at a public offering price of $47.50 per ordinary share, and, in lieu of ordinary shares to certain investors, Pre-Funded Warrants to purchase 0.5 million of the Company’s ordinary shares at the public offering price less a $0.0001 per ordinary share exercise price. The Company has authorized and reserved a sufficient number of ordinary shares to permit the full exercise of all outstanding Pre-Funded Warrants. The Pre-Funded Warrants are classified as a liability.

In connection with the recognition as taxable income of the $375.0 million upfront payment received in relation to the Royalty Financing Agreement, and the consumption of $30.3 million of losses carried forward, as well as the release of the valuation allowance related to these losses carried forward (refer to Note 14, “Income Taxes”), the Company allocated the tax benefit from the release of the valuation allowance related to net operating loss carryforwards generated by share issuance costs incurred in 2019 to additional paid-in capital. This resulted in an increase in additional paid-in capital of $0.9 million in the three and nine months ended September 30, 2025.

10Share-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 2025, the Company’s shareholders authorized an additional 2,400,000 shares for issuance under the 2014 Plan. As of September 30, 2025, a total of 4,414,685 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 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 September 30,				Nine months ended September 30,
	2025		2024		2025		2024

	(in thousands)				(in thousands)
Cost of contract manufacturing revenues	$	—	$	—	$	—	$	1,035
Research and development expenses		695		1,413		5,916		7,694
Selling, general and administrative expenses		2,827		2,025		7,100		8,780
Total	$	3,522	$	3,438	$	13,016	$	17,509

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


	Three months ended September 30,				Nine months ended September 30,
	2025		2024		2025		2024

	(in thousands)				(in thousands)
Award type/ESPP
Share options	$	1,829	$	2,003	$	5,128	$	7,576
Restricted share units		2,837		1,615		7,597		10,199
Performance share units		(1,159)		(180)		276		(276)
ESPP		15		—		15		10
Total	$	3,522	$	3,438	$	13,016	$	17,509

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As of September 30, 2025, the unrecognized share-based compensation expense related to unvested awards under the 2014 Plans were:


	Unrecognized		Weighted average
	share-based		remaining
	compensation		period for
	expense		recognition
	(in thousands)		(in years)
Award type
Share options	$	13,621	2.63
Restricted share units		20,501	2.23
Performance share units		166	0.25
Total	$	34,288	2.38

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 after one year from 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 nine months ended September 30, 2025:


	Options
	Number of	Weighted average
	ordinary shares	exercise price

Outstanding at December 31, 2024	4,967,160		19.87
Granted	1,220,410	$	12.63
Forfeited	(118,432)	$	13.17
Expired	(833,550)	$	29.86
Exercised	(553,556)	$	15.74
Outstanding at September 30, 2025	4,682,032	$	17.04
Thereof, fully vested, and exercisable on September 30, 2025	2,534,300	$	21.41
Thereof, outstanding and expected to vest after September 30, 2025	2,147,732	$	11.88
Outstanding and expected to vest after December 31, 2024	1,838,603	$	11.46


Total weighted average grant date fair value of options issued during the period (in $ millions)				$	9.4

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 September 30,		Nine months ended September 30,
	2025	2024	2025	2024
Assumptions
Expected volatility	80%	70%	70% - 80%	70%
Expected terms	10 years	10 years	10 years	10 years
Risk-free interest rate	4,38%	4,32%	4.36% - 4.64%	4,32%
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 nine months ended September 30, 2025:


	RSUs
		Weighted average
	Number of	grant-date fair
	ordinary shares	value
Non-vested at December 31, 2024	1,659,898	$	10.71
Granted	1,587,990	$	12.46
Vested	(747,388)	$	12.14
Forfeited	(213,147)	$	10.11
Non-vested at September 30, 2025	2,287,353	$	11.51

Total weighted average grant date fair value of RSUs granted during the period (in $ millions)		$	19.8

RSUs generally vest over one to three years. RSUs granted to non-executive directors vest one year from the date of grant.

PSUs

Awards of PSUs are subject to the achievement of specified performance objectives. The fair value of PSUs granted in 2022 and 2024 was determined on the grant date by reference to the market price of the Company’s ordinary shares.

The following table summarizes the PSU activity for the nine months ended September 30, 2025:


	PSUs
		Weighted average
	Number of	grant-date fair
	ordinary shares	value
Non-vested at December 31, 2024	150,120	$	21.65
Vested	(66,500)	$	26.70
Forfeited	(38,620)	$	20.24
Non-vested at September 30, 2025	45,000	$	15.40

ESPP

During the nine months ended September 30, 2025, nil ordinary shares were issued under the ESPP compared to 10,150 ordinary shares issued during the same period in 2024. As of September 30, 2025, 86,712 ordinary shares remain available for issuance under the ESPP compared to a total of 86,712 ordinary shares as of September 30, 2024.

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11	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 September 30,				Nine months ended September 30,
	2025		2024		2025		2024

	(in thousands)				(in thousands)
Revenue	$	3,701	$	2,287	$	10,530	$	21,898
Less:
Employee related expenses		(14,666)		(14,712)		(43,407)		(61,760)
Laboratory and development expenses		(18,675)		(9,028)		(50,235)		(27,508)
Professional fees		(4,814)		(1,792)		(9,995)		(6,751)
Facility expenses		(1,677)		(2,572)		(5,048)		(12,335)
Consumables		(750)		(1,439)		(2,383)		(8,123)
Other segment items⁽¹⁾		(2,418)		(2,185)		(5,397)		(10,248)
Segment operating loss		(39,299)		(29,441)		(105,935)		(104,827)
Reconciliation
Depreciation and amortization expense		(3,632)		(2,986)		(11,789)		(8,285)
Share-based compensation expense		(3,522)		(3,438)		(13,016)		(17,509)
Fair value loss - contingent consideration		(1,823)		411		(5,293)		2,032
Fair value loss - liability related to pre-funded warrants		(5,722)		—		(5,722)		—
Foreign currency gains, net		(428)		7,564		25,382		5,430
Interest income		3,503		4,866		11,154		17,179
Interest expense - Royalty Financing Agreement		(13,806)		(12,832)		(40,885)		(37,671)
Interest expense - Hercules debt facility		(1,794)		(3,779)		(5,415)		(11,194)
Other reconciling items		(5,379)		(4,774)		(818)		(9,877)
Consolidated loss before income tax expense	$	(71,903)	$	(44,409)	$	(152,338)	$	(164,722)

(1)

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

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12	CSL Behring collaboration

In June 2020, uniQure biopharma B.V. entered into the CSL Behring Agreement, pursuant to which CSL Behring received the exclusive global rights to HEMGENIX®.

Following the divestiture of the Company’s commercial manufacturing activities located in Lexington, MA in July 2024 (the “Lexington Transaction”), the Company determined that it is an agent in the sale of HEMGENIX® to CSL Behring. As a result of the Company being an agent, the Company recognizes corresponding costs related to the purchase of HEMGENIX® from Genezen Holdings Inc. and its affiliate Genezen MA, Inc. (together “Genezen”) net of income from the sales of HEMGENIX® to CSL Behring as well as income related to the release of liabilities associated with expected net losses in Other expense within the Company’s Consolidated Statements of Operations and Comprehensive Loss.

Accounts receivable related to collaboration services and royalty revenue were as follows:


	September 30,		December 31,
	2025		2024

	(in thousands)
Accounts receivable	$	3,883	$	5,881
Total	$	3,883	$	5,881

13	Other income

Other income for the three and nine months ended September 30, 2025 and 2024:


	Three months ended September 30,				Nine months ended September 30,
	2025		2024		2025		2024

	(in thousands)				(in thousands)
Sale of critical reagents to Genezen	$	—	$	—	$	6,000	$	—
Research and development grants from Dutch authorities		1,013		1,071		4,124		3,957
Sublease income		488		333		1,747		658
Gain on sale of commercial manufacturing activities		—		1,171		—		1,171
Other		9		16		542		164
Total	$	1,510	$	2,591	$	12,413	$	5,950

In the three and nine months ended September 30, 2025 the Company recognized nil and $6.0 million in income, respectively, related to a one-time sale of critical reagents to Genezen. There were no sales of critical reagents in the comparative period in 2024.

14	Income taxes

The effective income tax rate of 12.0% and 6.3% during the three and nine months ended September 30, 2025 (during the three and nine months ended September 30, 2024, (0.1%) and 1.0%, respectively) was 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.

Since 2023, the Company has been engaged with the Dutch tax authorities regarding the tax treatment of the $375.0 million upfront payment received in exchange for the rights to the lowest royalty tier on CSL Behring’s worldwide net sales of HEMGENIX®. Refer to Note 8, “Royalty Financing Agreement”. In September 2025, the Dutch tax authorities communicated their position that, for Dutch corporate income tax purposes, the upfront payment would be treated as taxable income in 2023. This would result in a taxable profit instead of a net operating loss for that year.

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On a provisional basis, the recognition of the $375.0 million upfront payment as taxable income resulted in the consumption of $30.3 million of losses carried forward, as well as the release of the associated valuation allowance. During the three and nine months ended September 30, 2025, the Company recorded, on a discrete basis, an estimate of $7.5 million of current tax expense related to the Dutch operations and $1.1 million of related interest expense, in accordance with ASC 740, Income Taxes. These estimates remain subject to change upon finalization of the 2023 filing position.

In connection with the estimate and associated release of the valuation allowance, the Company allocated part of such release to the tax benefit of share issuance costs incurred in 2019. This resulted in an increase in additional paid-in capital as well as deferred tax expenses of $0.9 million.

The following table summarizes the income tax (expense) / benefit for the three and nine months ended September 30, 2025 and 2024, respectively:


	Three months ended September 30,				Nine months ended September 30,
	2025		2024		2025		2024

	(in thousands)				(in thousands)
Current tax expense
Dutch operations	$	(7,479)	$	—	$	(7,479)	$	—
Other		(42)		(89)		(89)		(160)
Total current income tax expense	$	(7,521)	$	(89)	$	(7,568)	$	(160)
Deferred tax (expense) / benefit
Dutch operations	$	(860)	$	—	$	(860)	$	—
Other		(245)		120		(1,119)		(1,413)
Total deferred tax (expense) / benefit		(1,105)		120		(1,979)		(1,413)
Total income tax (expense) / benefit	$	(8,626)	$	31	$	(9,547)	$	(1,573)

15Basic and diluted loss per share

Diluted loss per 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 nine months ended September 30, 2025 and September 30, 2024, 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 nine months ended September 30, 2025 and September 30, 2024. 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 September 30, 2025 and September 30, 2024, respectively.

The potentially dilutive ordinary shares are summarized below:


	Three months ended September 30,				Nine months ended September 30,
	2025		2024		2025		2024
Anti-dilutive ordinary share equivalents
Stock options under 2014 Plans	$	4,682,032	$	5,425,575	$	4,682,032	$	5,425,575
Non-vested RSUs and PSUs		2,332,353		2,151,809		2,332,353		2,151,809
Pre-Funded Warrants		526,316		—		526,316		—
ESPP		7,239		—		7,239		—
Total anti-dilutive ordinary share equivalents		7,547,940		7,577,384		7,547,940		7,577,384

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16Subsequent events

As of September 30, 2025, the Company recorded its liability related to pre-funded warrants (refer to Note 5, “Fair value Measurement”) at a fair value of $30.7 million. The Company determined the fair value of the liability using the closing market price of its ordinary shares of $58.37 per ordinary share as of September 30, 2025. On November 3, 2025 the Company announced preliminary feedback from the FDA based on discussions at the Company’s pre-BLA meeting. Following such announcement, there was a decrease of the market price of the Company’s ordinary shares to $34.29 per ordinary share as of the close of the market on November 3, 2025. This reduced the fair value of the liability related to pre-funded warrants by $12.7 million to $18.0 million. The Company will determine the fair value of its liability related to pre-funded warrants based on the price of its ordinary shares at the close of the market on December 31, 2025. The Company will record the difference between the fair value as of September 30, 2025 and December 31, 2025 in income during the twelve month period ended December 31, 2025.

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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”), Fabry disease, and amyotrophic lateral sclerosis caused by mutations in superoxide dismutase 1 (“SOD1-ALS”).

Business Developments

Huntington’s disease program (AMT-130)

Clinical Data

We are conducting Phase I/II clinical trials of AMT-130 for the treatment of Huntington’s disease in the U.S. and Europe. The Phase I/II AMT-130 clinical trials were supplemented by two additional protocols, each with a statistical plan that was discussed with and submitted to the United States Food and Drug Administration (the “FDA”) earlier in 2025. The additional protocols, among other things, provide for the pooling of data across the ongoing U.S. and European studies, and also prespecified the pivotal comparison of AMT-130 clinical endpoints compared to a propensity score-matched external control from the Enroll-HD natural history data set.

In September 2025, we announced positive topline data from the pivotal Phase I/II study of AMT-130. The pivotal study met its prespecified primary endpoint, with high-dose AMT-130 demonstrating a statistically significant slowing of disease progression as measured by the composite Unified Huntington’s Disease Rating Scale (“cUHDRS”) at 36 months compared to a propensity score-matched external control. The study also met a key secondary endpoint by achieving statistically significant slowing of disease progression as measured by Total Functional Capacity (“TFC”) at 36 months compared to a propensity score-matched external control.

In accordance with the prospectively defined statistical analysis plan aligned with, and submitted, to the FDA, 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).

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

●

A statistically significant 75% slowing of disease progression as measured by 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 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 with no new drug-related serious adverse events observed since December 2022. The most common adverse events in the treatment groups were related to the administration procedure.

Data from the Phase I/II studies were presented at the 2025 Huntington’s Disease Clinical Research Congress, which took place from October 10-13, 2025, in Nashville, Tennessee.

In October 2025, we initiated and fully recruited a fourth cohort evaluating high-dose AMT-130 in six patients with lower striatal volumes compared to patients in previous cohorts. Patient dosing in the fourth cohort is expected to be completed before year-end 2025.

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Regulatory Update

In October 2025, we held a pre-Biologics License Application (“BLA”) meeting with the United States Food and Drug Administration (the “FDA”) to discuss the planned BLA submission for AMT-130. Though final meeting minutes have not yet been received, based on the discussions at the meeting, we believe that the FDA currently no longer agrees that data from the Phase I/II studies of AMT-130 in comparison to an external control, as per the prespecified protocols and statistical analysis plans shared with the FDA in advance of the analyses, may be adequate to provide the primary evidence in support of a BLA submission. Consequently, the timing of the BLA submission for AMT-130 is now unclear.

RMAT and Breakthrough Therapy Designations

In April 2025, we announced that the FDA granted Breakthrough Therapy designation to AMT-130 for the treatment of Huntington’s disease. The Breakthrough Therapy designation was supported by clinical data from the ongoing Phase I/II trials of AMT-130 for the treatment of Huntington’s disease showing the potential for meaningful slowing of disease progression. Breakthrough Therapy designation is intended to expedite the development and review of investigational therapeutic candidates that are intended to treat a serious condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy on a clinically significant endpoint(s). In general, the preliminary clinical evidence should show a clear advantage over available therapy.

In June 2024, we announced that the FDA granted us Regenerative Medicine Advanced Therapy (“RMAT”) designation for AMT-130 based on the potential of AMT-130 to address the major unmet medical need among patients with Huntington’s disease.

In addition to the RMAT and Breakthrough Therapy designations, the FDA previously granted Orphan Drug designation and Fast Track designation to AMT-130.

Financing

September 2025 Public offering

In September 2025, we received net proceeds of $323.7 million, after deducting underwriting discounts and commissions and other offering expenses payable by us, through a follow-on public offering of 6.7 million ordinary shares at a public offering price of $47.50 per ordinary share, and, in lieu of ordinary shares to certain investors, pre-funded warrants to purchase 0.5 million of our ordinary shares at the public offering price per share less the $0.0001 per share exercise price of each pre-funded warrant.

Hercules Loan Amendment

In September 2025, we entered into a $175.0 million senior secured term loan facility (the “2025 Amended Facility”) with Hercules Capital, Inc. (“Hercules”). The 2025 Amended Facility consists of three tranches including a first tranche of $50.0 million replacing the debt outstanding as per the loan amendment date, an additional term loan tranche of $100.0 million, which can be drawn at our option, subject to the BLA approval of AMT-130 prior to June 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, and a third tranche of up to $25.0 million, subject to Hercules’ approval. All tranches have a floating interest rate of the greater of 9.45% and the prime rate plus 2.45%, reflecting a floating rate of 9.70% as of September 30, 2025. The tranches mature on October 1, 2030.

January 2025 Public offering

In January 2025, we received net proceeds of $70.1 million, after deducting underwriting discounts and commissions and other offering expenses payable by us, through a follow-on public offering of 4.4 million ordinary shares at a public offering price of $17.00 per ordinary share. In February 2025, we received an additional $10.4 million in net proceeds, after related offering expenses, upon the underwriters’ exercise of their option to purchase an additional 0.7 million ordinary shares at the public offering price.

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Recent Developments of Other Product Candidates

Temporal lobe epilepsy program (AMT-260)

We are conducting a Phase I/IIa clinical trial (“GenTLE”) of AMT-260 for the treatment of MTLE in the United States. GenTLE consists of two parts. The first part is a multicenter, open-label trial with two dosing cohorts of six patients each to assess safety, tolerability, and first signs for efficacy of AMT-260 in patients with refractory MTLE. The second part is expected to be a randomized, controlled trial to generate proof of concept data.

In November 2024, we announced that the first patient had been dosed in GenTLE.

In September 2025, we completed enrollment of the first three patients in the first cohort, which, following a positive independent data monitoring committee (“IDMC”) review, allowed for the expansion of the first cohort into mesial temporal lobe epilepsy in the dominant hemisphere and the initiation of the second cohort.

Fabry disease program (AMT-191)

We are conducting a Phase I/IIa clinical trial of AMT-191 for the treatment of Fabry disease. The multicenter, open-label clinical trial consists of two dose-escalating cohorts of up to six adult male patients each to assess safety, tolerability, and early signs of efficacy of AMT-191 in patients with Fabry disease.

In August 2024, we announced that the first patient had been dosed. In September 2024, we announced that the FDA granted Orphan Drug designation to AMT-191 and in October 2024, the FDA granted Fast Track Designation to AMT-191.

In February 2025, we announced a favorable recommendation from the IDMC following a review of safety data from the first two patients treated in the Phase I/IIa study. The IDMC’s review did not identify any significant safety concerns and recommended proceeding with enrollment in the second cohort.

In September 2025, we announced initial safety and exploratory efficacy of the first cohort of our Phase I/IIa trial of AMT-191. We presented the preliminary data at the International Congress of Inborn Errors of Metabolism (“ICIEM”) in Kyoto, Japan in September 2025.

As of the July 24, 2025 study cutoff date, all patients in the first dose cohort showed sustained increases in α-galactosidase A (“α-Gal A”) enzyme activity, ranging from 27- to 208-fold above the mean normal range (1.38-8.66 nmol; mean normal of 3.57 nmol). All four patients were withdrawn from enzyme replacement therapy and maintained stable plasma lyso-Gb3 levels through the cutoff date. As of the July 24, 2025 study cutoff date, all patients had less than three months of follow-up.

Based on data observed to date, AMT-191 showed a manageable safety profile. At the high dose, two Serious Adverse Events (“SAEs”) unrelated to AMT-191 (stroke, diplopia), two related SAEs (chest pain, increased troponin), and one possibly related SAE (leptomeningeal enhancement) were observed in two patients. Additionally, one patient experienced an asymptomatic Grade 3 liver enzyme elevation that resolved with corticosteroid therapy. This event, classified as a dose-limiting toxicity per protocol, was not considered serious and did not require hospitalization. No loss of α-Gal A expression was observed in this patient.

A second, lower dose cohort of three patients completed enrollment and a third cohort of three patients is currently enrolling.

Amyotrophic Lateral Sclerosis (AMT-162)

EPISOD1 is a Phase I/II multi-center, open-label trial of AMT-162 for the treatment of SOD1-ALS being conducted in the United States consisting of three dose-escalating cohorts with up to four patients in each cohort. The trial will explore the safety and tolerability of AMT-162 and will assess exploratory signs of efficacy by measuring neurofilament light chain, a biomarker of neuronal damage, and SOD1 protein.

In October 2024, we announced the first patient had been dosed in EPISOD1. In January 2025, we announced that the IDMC had recommended proceeding with enrollment of the second cohort after a review of the 28-day safety data from the first cohort.

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During the third quarter of 2025, we voluntarily paused enrollment in EPISOD1 upon the recommendation of the IDMC following a September 2025 review of available preliminary data related to the safety and efficacy of AMT-162 in the context of a dose limiting toxicity, which resulted in an SAE determined to be related to AMT-162, that was observed in one patient in the second cohort. We continue to collect and evaluate data from the five patients dosed in EPISOD1 to date.

Financial Overview

Key components of our results of operations include the following:


	Three months ended September 30,				Nine months ended September 30,
	2025		2024		2025		2024

	(in thousands)				(in thousands)
Total revenues	$	3,701	$	2,287	$	10,530	$	21,898
Cost of license revenues		(398)		(264)		(1,251)		(648)
Cost of contract manufacturing revenues		—		(757)		—		(17,060)
Research and development expenses		(34,366)		(30,595)		(105,889)		(104,942)
Selling, general and administrative expenses		(19,438)		(11,575)		(43,846)		(41,279)
Net loss		(80,529)		(44,378)		(161,885)		(166,295)

As of September 30, 2025 and December 31, 2024, we had cash and cash equivalents and investment securities of $694.2 million and $367.5 million, respectively. We had a net loss of $80.5 million and $161.9 million in the three and nine months ended September 30, 2025, respectively, compared to a net loss of $44.4 million and $166.3 million for the same period in 2024. As of September 30, 2025 and December 31, 2024, we had accumulated deficits of $1,291.9 million and $1,130.0 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 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 nine months ended September 30, 2025.

Contract manufacturing

We entered into a development and commercial supply agreement with CSL Behring LLC (“CSL Behring”) in June 2020 for the commercial supply of etranacogene dezaparvovec-drlb (HEMGENIX®). In July 2024, we divested our commercial manufacturing activities located in Lexington, MA (the “Lexington Transaction”). Prior to the Lexington Transaction, we recognized contract manufacturing revenues related to the contract manufacturing of HEMGENIX® for CSL Behring and the cost to manufacture HEMGENIX® under such agreement as Cost of contract manufacturing revenues.

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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;

●

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 our acquisition of uniQure France SAS.

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 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, income from the subleasing of our Amsterdam facility and our Lexington, MA research and development facility, and for the nine months ended September 30, 2025, income from a one-time sale of critical reagents.

Following the Lexington Transaction, our expenses consist of costs incurred under our commercial supply agreement (“CSA”) with Genezen Holdings Inc. and its affiliate Genezen MA, Inc. (together “Genezen”). These include costs related to the purchase of HEMGENIX® from Genezen, net of income from the sales of HEMGENIX® to CSL Behring, amortization of the intangible asset for our favorable supply terms under the CSA and credits from the release of liabilities related to expected net losses associated with minimum purchase commitments under the 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 September 30, 2025 and 2024

The following table presents a comparison of our results of operations for the three months ended September 30, 2025 and 2024:


	Three months ended September 30,
	2025		2024		2025 vs 2024

	(in thousands)
Total revenues	$	3,701	$	2,287	$	1,414
Operating expenses:
Cost of license revenues		(398)		(264)		(134)
Cost of contract manufacturing revenues		—		(757)		757
Research and development expenses		(34,366)		(30,595)		(3,771)
Selling, general and administrative expenses		(19,438)		(11,575)		(7,863)
Total operating expenses		(54,202)		(43,191)		(11,011)
Other income		1,510		2,591		(1,081)
Other expense		(2,044)		(1,915)		(129)
Loss from operations		(51,035)		(40,228)		(10,807)
Non-operating expense, net		(20,868)		(4,181)		(16,687)
Net loss before income tax expense	$	(71,903)	$	(44,409)	$	(27,494)
Income tax expense		(8,626)		31		(8,657)
Net loss	$	(80,529)	$	(44,378)	$	(36,151)

Revenues

Our revenues for the three months ended September 30, 2025 and 2024 were as follows:


	Three months ended September 30,
	2025		2024		2025 vs 2024

	(in thousands)
License revenues	$	3,582	$	2,111	$	1,471
Collaboration revenues		119		176		(57)
Total revenues	$	3,701	$	2,287	$	1,414

License revenues

We recognize license revenues from CSL Behring related to royalty payments owed on HEMGENIX® sales, when earned. For the three months ended September 30, 2025 and September 30, 2024, we recognized $3.6 million and $2.1 million of license revenues, respectively.

Collaboration revenues

We provide services to CSL Behring in accordance with the CSL Behring Agreement. Collaboration revenues related to these contracted services is recognized when the related performance obligations are satisfied. For the three months ended September, 2025 and 2024, we recognized $0.1 million and $0.2 million of collaboration revenues from CSL Behring, respectively.

Cost of contract manufacturing revenues

Cost of contract manufacturing revenues in the three months ended September 30, 2025 were nil. In the three months ended September 30, 2024 we incurred $0.8 million in cost of contract manufacturing revenues related to the manufacture of HEMGENIX® prior to the Lexington Transaction in July 2024.

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R&D expense

R&D expenses for the three months ended September 30, 2025 were $34.4 million, compared to $30.6 million for the same period in 2024. 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 September 30,
	2025		2024		2025 vs 2024

	(in thousands)
Huntington's disease (AMT-130)	$	11,801	$	4,470	$	7,331
Temporal lobe epilepsy (AMT-260)		2,245		1,606		639
Fabry disease (AMT-191)		1,709		792		917
Amyotrophic lateral sclerosis (AMT-162)		1,638		910		728
Programs in preclinical development and platform related expenses		1,419		965		454
Total direct research and development expenses	$	18,812	$	8,743	$	10,069

Employee and contractor-related expenses		8,566		9,967		(1,401)
Facility expenses		3,716		3,839		(123)
Fair value changes related to contingent consideration		1,822		(410)		2,232
Share-based compensation expense		695		1,413		(718)
Disposables		492		1,013		(521)
Severance costs		—		3,378		(3,378)
Other expenses		263		2,652		(2,389)
Total other research and development expenses	$	15,554	$	21,852	$	(6,298)

Total research and development expenses	$	34,366	$	30,595	$	3,771

Direct research and development expenses

General

Prior to the Lexington Transaction, we supplied Good Manufacturing Practice (“GMP”) material utilized in our clinical programs for Huntington’s disease and Fabry disease as well as GMP material needed for our preclinical programs using our in-house manufacturing facility. The related expenses were part of Other research and development expenses. Following the closing of the Lexington Transaction, we rely on third party suppliers for these activities and present the associated expenses within Direct research and development expenses.

Huntington’s disease (AMT-130)

In the three months ended September 30, 2025 and September 30, 2024, we incurred costs of $11.8 million and $4.5 million respectively. The increase of $7.3 million includes $6.6 million of expenses incurred in relation to the preparation of the Biologics License Application submission for AMT-130 in the United States, with the remainder attributable to the execution of our U.S. and European studies.

Temporal lobe epilepsy (AMT-260)

In the three months ended September 30, 2025 and September 30, 2024, we incurred costs of $2.2 million and $1.6 million respectively, for the development of AMT-260. We incurred costs of $2.1 million and $0.1 million related to clinical trials and Chemistry, Manufacturing, and Controls (“CMC”) development, respectively, compared to costs of $1.0 million and $0.5 million in the comparative period.

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Fabry disease (AMT-191)

In the three months ended September 30, 2025 and September 30, 2024, we incurred costs of $1.7 million and $0.8 million, respectively, related to our development of AMT-191. We incurred costs of $1.5 million and $0.3 million related to clinical trials and CMC development, respectively, compared to costs of $0.7 million and $0.1 million in the comparative period.

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

In the three months ended September 30, 2025 and September 30, 2024, we incurred $1.6 million and $0.9 million of expenses, respectively, to initiate a Phase I/II clinical trial in which the first patient was enrolled in October 2024. We incurred $1.4 million and $0.2 million related to clinical trials and CMC development, respectively, compared to $0.7 million and $0.1 million in the comparative period.

Preclinical programs & platform development

In the three months ended September 30, 2025 and September 30 2024, we incurred $1.4 million and $1.0 million of costs, respectively, related primarily to our preclinical activities associated with product candidates for various other research programs and technology innovation projects.

Other research & development expenses

●

We incurred $8.6 million in personnel and contractor-related expenses in the three months ended September 30, 2025, compared to $10.0 million for the same period in 2024. The decrease was primarily related to the divestment of our commercial manufacturing activities in July 2024 as well as a reduction in expense following the August 2024 restructuring;

●

We incurred $3.7 million in operating and depreciation expenses related to our leased facilities in Amsterdam and Lexington, Massachusetts in the three months ended September 30, 2025, compared to $3.8 million in the same period in 2024;

●

We recognized a $1.8 million loss in the three months ended September 30, 2025, related to an increase in the fair value of contingent consideration associated with the acquisition of uniQure France SAS, compared to a $0.4 million gain for the same period in 2024;

●

We incurred $0.7 million in share-based compensation expenses in the three months ended September 30, 2025, compared to $1.4 million for the same period in 2024;

●

We incurred $0.5 million in disposables costs in the three months ended September 30, 2025, compared to $1.0 million for the same period in 2024;

●

We incurred nil severance costs in the three months ended September 30, 2025, compared to $3.4 million related to the Lexington Transaction for the same period in 2024; and,

●

We incurred $0.3 million of other expenses for the three months ended September 30, 2025, compared to $2.7 million for the same period in 2024.

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Selling, general and administrative expenses

Selling, general and administrative expenses for the three months ended September 30, 2025 were $19.4 million, compared to $11.6 million for the same period in 2024.


	Three months ended September 30,
	2025		2024		2025 vs 2024

	(in thousands)
Employee and contractor-related expenses	$	7,055	$	5,467	$	1,588
Professional fees		6,362		1,453		4,909
Share-based compensation expense		2,828		2,025		803
Intellectual property fees		582		505		77
Depreciation and facility costs		466		467		(1)
Information technology costs		310		201		109
Severance costs		—		740		(740)
Other expenses		1,835		717		1,118
Total selling, general and administrative expenses	$	19,438	$	11,575	$	7,863

●

We incurred $7.1 million in personnel and contractor-related expenses in the three months ended September 30, 2025, compared to $5.5 million in the same period in 2024. The increase was primarily the result of an increase in personnel and contractor-related expenses to support the preparation of a potential commercialization of AMT-130;

●

We incurred $6.4 million in professional fees in the three months ended September 30, 2025, compared to $1.5 million in the same period in 2024. The increase was primarily related to professional fees incurred in relation to the preparation of a potential commercialization of AMT-130;

●

We incurred $2.8 million in share-based compensation expenses in the three months ended September 30, 2025, compared to $2.0 million in the same period in 2024. The increase was primarily a result of an increase in awards granted in the three months ended September 30, 2025, in comparison to the prior period;

●

We incurred nil severance costs in the three months ended September 30, 2025, compared to $0.7 million related to the Lexington Transaction for the same period in 2024;

●

We incurred $1.8 million in other expenses in the three months ended September 30, 2025, compared to $0.7 million in the same period in 2024.

Other items, net

Our other items, net, for the three months ended September 30, 2025 and September 30, 2024 were as follows:


	Three months ended September 30,
	2025		2024		2025 vs 2024

	(in thousands)
Research and development grants from Dutch authorities	$	1,012	$	1,071	$	(59)
Gain on sale of commercial manufacturing activities		—		1,171		(1,171)
Sublease income / (expense), net		31		(115)		146
Supply of HEMGENIX® to CSL Behring		(1,586)		(1,467)		(119)
Other income, net		9		16		(7)
Total other items, net	$	(534)	$	676	$	(1,210)

We recognized $1.6 million and $1.5 million in other expenses during the three months ended September 30, 2025 and September 30 2024, respectively, related to the purchase of HEMGENIX® from Genezen, net of income from the sales of HEMGENIX® to CSL Behring, amortization of the intangible asset for our favorable supply terms under the CSA and credits from the release of liabilities related to expected net losses associated with minimum purchase commitments under the CSA.

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Other non-operating items, net

Our other non-operating items, net, for the three months ended September 30, 2025 and September 30, 2024 were as follows:


	Three months ended September 30,
	2025		2024		2025 vs 2024

	(in thousands)
Interest income	$	3,503	$	4,866

uniQure N.V. 10-Q