uniQure (QURE) 10-Q 7 May 24 2024 Q1 Quarterly report Financial data

Document and Entity Information

Document and Entity Information - shares	3 Months Ended
	Mar. 31, 2024	May 02, 2024
Document and Entity Information
Document Type	10-Q
Document Quarterly Report	true
Document Period End Date	Mar. 31, 2024
Document Transition Report	false
Entity File Number	001-36294
Entity Registrant Name	uniQure N.V.
Entity Incorporation, State or Country Code	P7
Entity Tax Identification Number	00-0000000
Entity Address, Address Line One	Paasheuvelweg 25
Entity Address, City or Town	Amsterdam
Entity Address, Country	NL
Entity Address, Postal Zip Code	1105 BP
City Area Code	31
Local Phone Number	20-240-6000
Title of 12(b) Security	Ordinary Shares, par value €0.05
Trading Symbol	QURE
Security Exchange Name	NASDAQ
Entity Current Reporting Status	Yes
Entity Interactive Data Current	Yes
Entity Filer Category	Accelerated Filer
Entity Small Business	false
Entity Emerging Growth Company	false
Entity Shell Company	false
Entity Common Stock, Shares Outstanding		48,549,437
Entity Central Index Key	0001590560
Current Fiscal Year End Date	--12-31
Document Fiscal Year Focus	2024
Document Fiscal Period Focus	Q1
Amendment Flag	false

UNAUDITED CONSOLIDATED BALANCE

UNAUDITED CONSOLIDATED BALANCE SHEETS - USD ($) $ in Thousands	Mar. 31, 2024	Dec. 31, 2023
Current assets
Cash and cash equivalents	$ 243,062	$ 241,360
Current investment securities	312,621	376,532
Accounts receivable	10,717	4,193
Inventories, net	7,672	12,024
Prepaid expenses	18,839	15,089
Other current assets and receivables	3,092	2,655
Total current assets	596,003	651,853
Non-current assets
Property, plant and equipment, net of accumulated depreciation of $57.8 million as of March 31, 2024 and $55.7 million as of December 31, 2023	44,554	46,548
Operating lease right-of-use assets	27,695	28,789
Intangible assets, net, including in-process research and development asset of $57.8 million as of March 31, 2024 and $59.1 million as of December 31, 2023	59,111	60,481
Goodwill	25,795	26,379
Deferred tax assets, net	11,594	12,276
Other non-current assets	5,298	5,363
Total non-current assets	174,047	179,836
Total assets	770,050	831,689
Current liabilities
Accounts payable	5,231	6,586
Accrued expenses and other current liabilities	22,658	30,534
Current portion of contingent consideration	27,587	28,211
Current portion of operating lease liabilities	7,997	8,344
Total current liabilities	63,473	73,675
Non-current liabilities
Long-term debt	102,120	101,749
Liability from royalty financing agreement	405,398	394,241
Operating lease liabilities, net of current portion	26,983	28,316
Contingent consideration, net of current portion	14,625	14,795
Deferred tax liability, net	7,376	7,543
Other non-current liabilities	3,321	3,700
Total non-current liabilities	559,823	550,344
Total liabilities	623,296	624,019
Commitments and contingencies
Shareholders' equity
Ordinary shares, €0.05 par value: 80,000,000 shares authorized as of March 31, 2024 and December 31, 2023 and 48,492,357 and 47,833,830 ordinary shares issued and outstanding as of March 31, 2024 and December 31, 2023, respectively	2,919	2,883
Additional paid-in-capital	1,155,904	1,148,749
Accumulated other comprehensive loss	(56,042)	(53,553)
Accumulated deficit	(956,027)	(890,409)
Total shareholders' equity	146,754	207,670
Total liabilities and shareholders' equity	$ 770,050	$ 831,689

UNAUDITED CONSOLIDATED BALANC_2

UNAUDITED CONSOLIDATED BALANCE SHEETS (Parenthetical) $ in Thousands	Mar. 31, 2024 USD ($) shares	Mar. 31, 2024 € / shares	Dec. 31, 2023 USD ($) shares	Dec. 31, 2023 € / shares
Accumulated depreciation | $	$ 57,800		$ 55,700
Intangible assets, net | $	$ 59,111		$ 60,481
Ordinary shares, par value (in euros per share) | € / shares		€ 0.05		€ 0.05
Ordinary shares, authorized | shares	80,000,000		80,000,000
Ordinary shares, issued | shares	48,492,357		47,833,830
Ordinary shares, outstanding | shares	48,492,357		47,833,830
In-process research & development
Intangible assets, net | $	$ 57,800		$ 59,100

UNAUDITED CONSOLIDATED STATEMEN

UNAUDITED CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2024	Mar. 31, 2023
Total revenues	$ 8,485	$ 5,325
Operating expenses:
Cost of license revenues	(150)
Cost of contract manufacturing revenues	(9,076)	(2,435)
Research and development expenses	(40,692)	(60,809)
Selling, general and administrative expenses	(13,937)	(17,848)
Total operating expenses	(63,855)	(81,092)
Other income	1,376	1,811
Other expense	(234)	(216)
Loss from operations	(54,228)	(74,172)
Interest income	6,508	1,669
Interest expense	(16,097)	(3,562)
Foreign currency losses, net	(1,145)	(2,369)
Loss before income tax (expense) / benefit	(64,962)	(78,434)
Income tax (expense) / benefit	(656)	1,207
Net loss	(65,618)	(77,227)
Other comprehensive (loss) / income:
Foreign currency translation (loss) / gains, net	(2,524)	5,797
Defined benefit pension gain, net of taxes	35
Total comprehensive loss	$ (68,107)	$ (71,430)
Earnings per ordinary share - basic
Basic net (loss) / income per ordinary share	$ (1.36)	$ (1.63)
Earnings per ordinary share - diluted
Diluted net (loss) / income per ordinary share	$ (1.36)	$ (1.63)
Weighted average shares - basic	48,384,510	47,436,335
Weighted average shares - diluted	48,384,510	47,436,335
License revenues
Total revenues	$ 1,202	$ 0
Contract manufacturing revenues
Total revenues	3,990	4,937
Collaboration revenues
Total revenues	$ 3,293	$ 388

UNAUDITED CONSOLIDATED STATEM_2

UNAUDITED CONSOLIDATED STATEMENTS OF SHAREHOLDERS' EQUITY - USD ($) $ in Thousands	Ordinary shares	Additional paid-in capital	Accumulated other comprehensive income /(loss)	Accumulated deficit	Total
Beginning balance at Dec. 31, 2022	$ 2,838	$ 1,113,393	$ (58,291)	$ (581,931)	$ 476,009
Beginning balance (in shares) at Dec. 31, 2022	46,968,032
Increase (decrease) in shareholders' equity
Loss for the period				(77,227)	(77,227)
Other comprehensive income (loss), net			5,797		5,797
Exercises of share options	$ 1	86			87
Exercises of share options (in shares)	10,055
Restricted and performance share units distributed during the period	$ 30	(30)
Restricted and performance share units distributed during the period (in shares)	566,091
Share-based compensation expense		8,061			8,061
Issuance of ordinary shares relating to employee stock purchase plan		44			44
Issuance of ordinary shares relating to employee stock purchase plan (in shares)	2,495
Ending balance at Mar. 31, 2023	$ 2,869	1,121,554	(52,494)	(659,158)	412,771
Ending balance (in shares) at Mar. 31, 2023	47,546,673
Beginning balance at Dec. 31, 2023	$ 2,883	1,148,749	(53,553)	(890,409)	$ 207,670
Beginning balance (in shares) at Dec. 31, 2023	47,833,830				47,833,830
Increase (decrease) in shareholders' equity
Loss for the period				(65,618)	$ (65,618)
Other comprehensive income (loss), net			(2,489)		(2,489)
Restricted and performance share units distributed during the period	$ 36	(36)
Restricted and performance share units distributed during the period (in shares)	658,527
Share-based compensation expense		7,191			7,191
Ending balance at Mar. 31, 2024	$ 2,919	$ 1,155,904	$ (56,042)	$ (956,027)	$ 146,754
Ending balance (in shares) at Mar. 31, 2024	48,492,357				48,492,357

UNAUDITED CONSOLIDATED STATEM_3

UNAUDITED CONSOLIDATED STATEMENTS OF CASH FLOWS - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2024	Mar. 31, 2023
Cash flows from operating activities
Net loss	$ (65,618)	$ (77,227)
Adjustments to reconcile net loss to net cash used in operating activities:
Depreciation and amortization	2,629	2,527
Amortization of discount on investment securities	(3,695)	(925)
Share-based compensation expense	7,191	8,061
Royalty financing agreement interest expense	12,362
Deferred tax expense / (income)	656	(1,207)
Changes in fair value of contingent consideration	165	975
Unrealized foreign exchange losses, net	1,389	1,193
Other items, net	2,778	456
Changes in operating assets and liabilities:
Accounts receivable, prepaid expenses, and other current assets and receivables	(10,417)	(936)
Inventories	2,236	(553)
Accounts payable	(701)	(1,661)
Accrued expenses, other liabilities, and operating leases	(9,550)	(9,005)
Net cash used in operating activities	(60,575)	(78,302)
Cash flows from investing activities
Proceeds on maturity of debt securities	150,107	5,330
Investment in debt securities	(83,778)
Purchases of property, plant, and equipment	(2,344)	(2,342)
Net cash generated from investing activities	63,985	2,988
Cash flows from financing activities
Proceeds from issuance of ordinary shares related to employee stock option and purchase plans		131
Net cash generated from financing activities		131
Currency effect on cash, cash equivalents and restricted cash	(1,725)	1,034
Net increase / (decrease) in cash, cash equivalents and restricted cash	1,685	(74,149)
Cash, cash equivalents and restricted cash at beginning of period	244,544	231,173
Cash, cash equivalents and restricted cash at the end of period	246,229	157,024
Cash and cash equivalents	243,062	153,851
Restricted cash related to leasehold and other deposits	3,167	3,173
Total cash, cash equivalents and restricted cash	246,229	157,024
Supplemental cash flow disclosures:
Cash paid for interest	(4,761)	(3,057)
Non-cash decrease in accounts payables and accrued expenses and other current liabilities related to purchases of property, plant, and equipment	$ (577)	$ (753)

General business information

General business information	3 Months Ended
	Mar. 31, 2024
General business information
General business information	1 ​ uniQure N.V. (the “Company”) was incorporated on January 9, 2012, as a private company with limited liability ( besloten vennootschap met beperkte aansprakelijkheid naamloze vennootschap ​ The Company is registered in the trade register of the Dutch Chamber of Commerce ( Kamer van Koophandel ​ The Company’s ordinary shares are listed on the Nasdaq Global Select Market and trade under the symbol “QURE”.

Summary of significant accounti

Summary of significant accounting policies	3 Months Ended
	Mar. 31, 2024
Summary of significant accounting policies
Summary of significant accounting policies	2 Summary of significant accounting policies ​ 2.1 Basis 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 Update (“ASU”) of the Financial Accounting Standards Board (“FASB”). ​ 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. ​ 2.2 Unaudited 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 months ended March 31, 2024, are not necessarily indicative of the results to be expected for the full year ending December 31, 2024, 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 ​ 2.3 Use 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 assets and liabilities at the date of the financial statements and reported amounts of revenues and expenses during the reporting period. Actual results could differ from those estimates. ​ 2.4 Accounting 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, 2023, and the notes thereto, which are included in the Annual Report ​ 2.5 Recent accounting pronouncements ​ There have been no new accounting pronouncements or changes to accounting pronouncements during the three months ended March 31, 2024, as compared to the recent accounting pronouncements described in Note 2.3.27 of the Annual Report ​

CSL Behring collaboration

CSL Behring collaboration	3 Months Ended
	Mar. 31, 2024
CSL Behring collaboration
CSL Behring collaboration	3 CSL Behring collaboration ​ On June 24, 2020, uniQure biopharma B.V. entered into a commercialization and license agreement with CSL Behring (the “CSL Behring Agreement”), pursuant to which CSL Behring received exclusive global rights to HEMGENIX® ​ The transaction became fully effective on May 6, 2021. ​ License revenue The Company recognized $1.2 million of royalty revenue in the three months ended , compared to nil in the three months ended . Royalties on the sale of HEMGENIX® are recorded once earned and are presented as license revenue. Accounts receivable and contract asset ​ As of December 31, 2023, the Company recorded accounts receivable of $4.0 million from CSL Behring related to collaboration services, contract manufacturing revenue and royalty revenue. ​ As of , the Company had accounts receivable of $10.6 million from CSL Behring related to collaboration services, contract manufacturing revenue and royalty revenue. ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​

Investment securities

Investment securities	3 Months Ended
	Mar. 31, 2024
Investment securities
Investment securities	4 Investment securities The following tables summarize the Company’s investments in sovereign debt as of March 31, 2024 and December 31, 2023: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ At March 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) ​ $ 312,621 ​ $ 12 ​ $ — ​ $ 312,633 Total ​ $ 312,621 ​ $ 12 ​ $ — ​ $ 312,633 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ At December 31, 2023 ​ ​ Amortized cost ​ Gross unrealized holding gains ​ Gross unrealized holding losses ​ Estimated fair value ​ ​ (in thousands) Current investments: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Government debt securities (held-to-maturity) ​ $ 376,532 ​ $ 139 ​ $ — ​ $ 376,671 Total ​ $ 376,532 ​ $ 139 ​ $ — ​ $ 376,671 ​ The Company invests in short-term U.S. and European government bonds with the highest investment credit rating. The U.S. and European government bonds are U.S. dollar and euro denominated, respectively. ​ Inputs to the fair value of the investments are considered Level 2 inputs.

Inventories, net

Inventories, net	3 Months Ended
	Mar. 31, 2024
Inventories, net
Inventories, net	5 Inventories, net ​ The following table summarizes the inventories, net balances as of March 31, 2024 and December 31, 2023: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ March 31, ​ December 31, ​ 2024 2023 ​ ​ (in thousands) Raw materials ​ $ 6,331 ​ $ 7,157 Work in progress ​ ​ 1,341 ​ ​ 4,109 Finished goods ​ ​ — ​ ​ 758 Inventories ​ $ 7,672 ​ $ 12,024 ​ The Company recorded write downs to net realizable value of $1.8 million for the three months ended and nil for the same period in 2023. The costs are recognized as Cost of Contract Manufacturing Revenues.

Fair value measurement

Fair value measurement	3 Months Ended
	Mar. 31, 2024
Fair value measurement
Fair value measurement	6 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 Measurements and Disclosures ​ 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 consolidated balance sheets approximate their fair values due to their short-term maturities. ​ The following table sets forth the Company’s assets and liabilities that are required to be measured at fair value on a recurring basis as of March 31, 2024 and December 31, 2023 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ 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) ​ ​ At December 31, 2023 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Assets: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Cash and cash equivalents ​ $ 241,360 ​ $ — ​ $ — ​ $ 241,360 ​ Cash and cash equivalents Restricted cash ​ ​ 3,184 ​ ​ — ​ ​ — ​ ​ 3,184 ​ Other non-current assets Total assets ​ $ 244,544 ​ $ — ​ $ — ​ $ 244,544 ​ ​ Liabilities: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Contingent consideration ​ ​ — ​ ​ — ​ ​ 43,006 ​ ​ 43,006 ​ Contingent consideration Consideration for post-acquisition services ​ ​ — ​ ​ — ​ ​ 457 ​ ​ 457 ​ Other non-current liabilities Total liabilities ​ $ — ​ $ — ​ $ 43,463 ​ $ 43,463 ​ ​ At March 31, 2024 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Assets: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Cash and cash equivalents ​ $ 243,062 ​ $ — ​ $ — ​ $ 243,062 ​ Cash and cash equivalents Restricted cash ​ ​ 3,167 ​ ​ — ​ ​ — ​ ​ 3,167 ​ Other non-current assets Total assets ​ $ 246,229 ​ $ — ​ $ — ​ $ 246,229 ​ ​ Liabilities: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Contingent consideration ​ ​ — ​ ​ — ​ ​ 42,212 ​ ​ 42,212 ​ Contingent consideration Consideration for post-acquisition services ​ ​ — ​ ​ — ​ ​ 498 ​ ​ 498 ​ Other non-current liabilities Total liabilities ​ $ — ​ $ — ​ $ 42,710 ​ $ 42,710 ​ ​ ​ Contingent consideration ​ The Company is required to pay up to EUR 178.8 million (or $193.0 million based on the foreign exchange rate on March 31, 2024) to the former shareholders of uniQure France SAS (formerly Corlieve Therapeutics SAS) upon the achievement of contractually defined milestones in connection with the Company’s July 2021 acquisition of uniQure France SAS. ​ The fair value of the contingent consideration as of March 31, 2024 was $42.2 million (December 31, 2023: $43.0 million) using discount rates of approximately 14.8% to 15.6% (December 31, 2023: 15.3% to 15.6%). The Company assumes the probability of achieving a EUR 30.0 million ($32.4 million) milestone payment following the dosing of the first patient in Phase I/II clinical trial to be 100%. ​ If as of March 31, 2024 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 $74.8 million. If as of March 31, 2024 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 contingent consideration between December 31, 2023 and March 31, 2024: ​ ​ ​ ​ ​ ​ Amount of ​ contingent ​ consideration ​ ​ 2024 ​ (in thousands) Balance at December 31, 2023 $ 43,006 Change in fair value (presented within research and development expenses) ​ 165 Currency translation effects ​ (959) Balance at March 31, 2024 $ 42,212 ​ As of March 31, 2024, the Company classified $27.6 million (December 31, 2023: $28.2 million) of the total contingent consideration of $42.2 million (December 31, 2023: $43.0 million) as current liabilities. The balance sheet classification 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

Accrued expenses and other curr

Accrued expenses and other current liabilities	3 Months Ended
	Mar. 31, 2024
Accrued expenses and other current liabilities
Accrued expenses and other current liabilities	7 Accrued expenses and other current liabilities ​ Accrued expenses and other current liabilities include the following items: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ March 31, ​ December 31, ​ 2024 2023 ​ ​ (in thousands) Personnel related accruals and liabilities ​ $ 9,572 ​ $ 16,263 Accruals for goods received from and services provided by vendors-not yet billed ​ ​ 11,881 ​ ​ 12,834 Liability owed to the Purchaser pursuant to the Royalty Financing Agreement ​ ​ 1,205 ​ ​ 1,437 Total ​ $ 22,658 ​ $ 30,534 ​

Long-term debt

Long-term debt	3 Months Ended
	Mar. 31, 2024
Long-term debt
Long-term debt	8 Long-term debt ​ On June 14, 2013, the Company entered into a venture debt loan facility with Hercules Capital, Inc. (formerly known as Hercules Technology Growth Capital, Inc.) (“Hercules”). The facility was amended and restated in 2014, 2016, 2018, January 2021, December 2021 (the “2021 Restated Facility”) and on May 12, 2023 (the “2023 Amended Facility”). ​ The total principal outstanding under the 2023 Amended Facility is $100.0 million. ​ The 2023 Amended Facility extends the maturity date and interest-only period from December 1, 2025 to January 5, 2027 (the “Maturity Date”). ​ The Company is required to repay the entire principal balance on the Maturity Date. The interest rate is adjustable and is the greater of (i) 7.95% and (ii) 7.95% plus the prime rate The amortized cost (including interest due presented as part of accrued expenses and other current liabilities) of the 2023 Amended Facility was $103.3 million as of March 31, 2024, compared to $102.9 million as of December 31, 2023, and is recorded net of discount and debt issuance costs. The foreign currency loss on the facility in the three months ended March 31, 2024 was $2.3 million, compared to a foreign currency gain of $0.7 million during the same period in 2023. Interest expense associated with the 2023 Amended Facility during the three months ended March 31, 2024 was $3.7 million, compared to $3.6 million during the same period in 2023. Under the 2023 Amended Facility the Company must remain current in its periodic reporting requirements and is required to keep a minimum cash balance deposited in bank accounts in the U.S. equivalent to the lesser of (i) 65% of the outstanding balance of principal due or (ii) 100% of worldwide cash and cash equivalents. This restriction on cash and cash equivalents only relates to the location of the cash and cash equivalents, and such cash and cash equivalents can be used at the discretion of the Company. Beginning on April 1, 2024, the Company is required to keep a minimum of unrestricted cash equal to at least 30% of the loan amount outstanding. In combination with other covenants, the 2023 Amended Facility restricts the Company’s ability to, among other things, incur future indebtedness and obtain additional debt financing, to make investments in securities or in other companies, to transfer assets, to perform certain corporate changes, to make loans to employees, officers, and directors, and to make dividend payments and other distributions to its shareholders. The Company secured the facilities by directly or indirectly pledging its total assets of $770.1 million, less $6.8 million of cash and cash equivalents and other current assets held by the Company, and $87.7 million of other current assets and investment held by uniQure France SAS as well as receivables sold to the Purchaser. Under the 2023 Amended Facility, the occurrence of a material adverse effect, as defined therein, would entitle Hercules to declare all principal, interest and other amounts owed by the Company immediately due and payable. As of March 31, 2024, the Company was in material compliance with all covenants and provisions.

Royalty Financing Agreement

Royalty Financing Agreement	3 Months Ended
	Mar. 31, 2024
Royalty Financing Agreement
Royalty Financing Agreement	9 ​ On May 12, 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’s worldwide net sales of HEMGENIX® for certain current and future royalties due to the Company. The Company is also eligible to receive an additional $25.0 million milestone payment under the Royalty Financing Agreement if 2024 net sales of HEMGENIX® exceed a pre-specified threshold, as set forth in the Royalty Financing Agreement. The Purchaser will receive 1.85 times the upfront payment (or $693.8 million) and 1.85 times the $25.0 million milestone payment (if paid) 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 of the upfront and milestone payment (if paid) through December 31, 2038. If 2024 net sales do not exceed a pre-specified threshold, the Company will be obligated to pay $25.0 million to the Purchaser but only to the extent that the Company achieves a future sales milestone under the CSL Behring Agreement. If such milestone payment is not due from CSL Behring, the Company is not obligated to pay any amounts to the Purchaser. 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 CSL Behring Agreement. Net proceeds from the Royalty Financing Agreement, after deducting professional and financial advisory fees related to the transaction of $4.9 million, were $370.1 million. The Company initially recorded these net proceeds as “Liability from royalty financing agreement” at their fair market value on its balance sheet as of closing of the transaction on June 5, 2023. Following the initial recognition, the Company records the debt at amortized cost. The Company expects to satisfy its commitment to the Purchaser prior to the First Hard Cap Date. The Company will record the difference of $323.7 million between the total expected payments of $693.8 million to the Purchaser and the $370.1 million net proceeds as interest expense using the effective interest rate method. The Company determined the effective interest rate based on the projected cash flows up to the First Hard Cap Date. Based on the Company’s projections the effective interest rate is expected to be within a range of 12.0% per annum to 13.5% per annum. The Company would have recorded between $12.1 million and $13.7 million of interest expense through the three months ended March 31, 2024 (nil for the three months ended March 31, 2023) if it had used 12.0% or 13.5%, instead of the $12.4 million recorded in the three months ended March 31, 2024 (nil for the three months ended March 31, 2023). The Company will prospectively update the effective interest rate at each reporting date based on updated projections. ​ The liability was initially recognized at fair value and inputs were considered Level 3 inputs. ​ ​ ​ The following table presents the movement in the liability related to the Royalty Financing Agreement between the December 31, 2023 and March 31, 2024: ​ ​ ​ ​ ​ ​ ​ ​ Amount of liability ​ ​ ​ (in thousands) Balance as of December 31, 2023 (includes $1.4 million presented as "Accrued expenses and other current liabilities) ​ $ 395,678 Royalty payments to Purchaser ​ ​ (1,437) Liability owed to the Purchaser (presented as "Accrued expense and other current liabilities") ​ ​ (1,205) Interest expense for the period ​ ​ 12,362 Liability related to the royalty financing agreement ​ $ 405,398 ​

Share-based compensation

Share-based compensation	3 Months Ended
	Mar. 31, 2024
Share-based compensation
Share-based compensation	10 ​ The Company’s share-based compensation plans include the 2014 Amended and Restated Share Option Plan (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”). The number of shares authorized for issuance under the 2014 Plan is 14 ,351,471 . ​ In June 2018, the Company’s shareholders adopted and approved an employee share purchase plan (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 85% of the lower of the closing market price on the offering date and the closing market price on the purchase date of each three-month offering period. ​ 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 March 31, ​ ​ 2024 ​ 2023 ​ ​ (in thousands) Cost of manufacturing services revenue ​ $ 345 ​ $ 24 Research and development ​ ​ 3,425 ​ ​ 4,305 Selling, general and administrative ​ ​ 3,421 ​ ​ 3,732 Total ​ $ 7,191 ​ $ 8,061 ​ Share-based compensation expense recognized by award type for the 2014 Plans as well as the ESPP was as follows: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three months ended March 31, ​ ​ 2024 ​ 2023 ​ ​ (in thousands) Award type/ESPP ​ ​ ​ ​ ​ ​ Share options ​ $ 2,913 ​ $ 3,274 Restricted share units ​ ​ 4,370 ​ ​ 4,630 Performance share units ​ ​ (99) ​ ​ 150 Employee share purchase plan ​ ​ 7 ​ ​ 7 Total ​ $ 7,191 ​ $ 8,061 ​ As of March 31, 2024, 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 ​ $ 20,743 ​ 2.56 Restricted share units ​ ​ 27,981 ​ 1.99 Performance share units ​ ​ 80 ​ 0.61 Total ​ $ 48,804 ​ 2.23 ​ 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 tables summarize option activity under the 2014 Plans for the three months ended March 31, 2024: ​ ​ ​ ​ ​ ​ ​ ​ ​ Options ​ ​ Number of ​ Weighted average ​ ordinary shares exercise price ​ ​ ​ ​ ​ ​ Outstanding at December 31, 2023 ​ 4,974,030 ​ ​ 23.25 Granted ​ 930,900 ​ $ 5.59 Forfeited ​ (71,437) ​ $ 21.26 Expired ​ (11,584) ​ $ 37.61 Outstanding at March 31, 2024 ​ 5,821,909 ​ $ 20.42 Thereof, fully vested, and exercisable on March 31, 2024 ​ 3,054,242 ​ $ 25.70 Thereof, outstanding and expected to vest after March 31, 2024 ​ 2,767,667 ​ $ 14.60 Outstanding and expected to vest after December 31, 2023 ​ 2,098,557 ​ $ 23.38 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Total weighted average grant date fair value of options issued during the period (in $ millions) ​ ​ ​ $ 3.0 ​ The fair value of each option issued is estimated at the respective grant date using the Hull & White option pricing model with the following weighted-average assumptions: ​ ​ ​ ​ ​ ​ ​ ​ Three months ended March 31, Assumptions 2024 2023 Expected volatility ​ 70% ​ 70% Expected terms ​ 10 years ​ 10 years Risk free interest rate ​ 4.32% ​ 4.10% Expected dividend yield ​ 0% ​ 0% ​ RSUs ​ The following table summarizes the RSU activity for the three months ended March 31, 2024: ​ ​ ​ ​ ​ ​ ​ ​ ​ RSUs ​ ​ Weighted average ​ ​ Number of ​ grant-date fair ​ ​ ordinary shares ​ value Non-vested at December 31, 2023 ​ 2,264,369 ​ $ 18.07 Granted ​ 1,160,800 ​ $ 5.59 Vested ​ (659,240) ​ $ 20.30 Forfeited ​ (58,401) ​ $ 17.39 Non-vested at March 31, 2024 ​ 2,707,528 ​ $ 12.19 ​ ​ ​ ​ ​ ​ Total weighted average grant date fair value of RSUs granted during the period (in $ millions) ​ ​ ​ $ 6.5 ​ RSUs generally vest over one ​ PSUs ​ The following table summarizes the PSU activity for the three months ended March 31, 2024: ​ ​ ​ ​ ​ ​ ​ ​ ​ PSUs ​ ​ Weighted average ​ ​ Number of ​ grant-date fair ​ ​ ordinary shares ​ value Non-vested at December 31, 2023 ​ 222,550 ​ $ 28.09 Forfeited ​ (17,030) ​ $ 27.12 Non-vested at March 31, 2024 ​ 205,520 ​ $ 27.99 ​ The Company granted ordinary shares to certain employees in December 2021 and at various dates during the year ended December 31, 2022 that will be earned upon achievement of defined milestones. Earned ordinary shares will vest upon the later of a minimum service period of one year or three years, or the achievement of defined milestones, subject to the grantee’s continued employment. In addition, portions of the ordinary shares granted in December 2021 to executives and other members of senior management are subject to achieving a minimum total shareholder return relative to the NASDAQ Biotechnology Index. The Company recognizes the compensation cost related to these grants to the extent it considers achievement of the milestones to be probable. As of March 31, 2024, two milestones had been achieved and vested in either 2022 or 2023. Additionally, another two milestones are considered probable as of December 31, 2023 and March 31, 2024. ​ The ESPP ​ During the three months ended March 31, 2024, nil ordinary shares were issued under the ESPP compared to 2,495 during the same period in 2023. As of March 31, 2024, 96,862 ordinary shares remain available for issuance under the ESPP compared to a total of 113,565 as of March 31, 2023.

Income taxes

Income taxes	3 Months Ended
	Mar. 31, 2024
Income taxes
Income taxes	11 Income taxes ​ The Company recorded $0.7 million deferred tax expense in relation to its operations in the U.S. during the three months ended March 31, 2024. The Company recorded $1.2 million deferred tax benefit in relation to its operations in the U.S. and France during the three months ended March 31, 2023. ​ The effective income tax rate of 1.0% during the three ended March 31, 2024 is substantially lower than the enacted rate of 25.8% in the Netherlands as the Company records a valuation allowance against its net deferred tax assets in the Netherlands and a partial a valuation allowance against its net deferred tax assets in France. The effective income tax rate during the three months ended March 31, 2023 was (1.5%), as the Company had recorded a valuation allowance against its net deferred tax assets in the Netherlands. ​ ​

Basic and diluted earnings per

Basic and diluted earnings per share	3 Months Ended
	Mar. 31, 2024
Basic and diluted earnings per share
Basic and diluted earnings per share	14 Basic and diluted earnings per share ​ ​ The potentially dilutive ordinary shares are summarized below: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three months ended March 31, ​ ​ 2024 ​ 2023 Anti-dilutive ordinary share equivalents ​ ​ ​ ​ ​ ​ Stock options under 2014 Plans and previous plan ​ ​ 5,821,909 ​ ​ 5,150,690 Non-vested RSUs and PSUs ​ ​ 2,913,048 ​ ​ 2,772,146 ESPP ​ ​ 3,140 ​ ​ 801 Total anti-dilutive ordinary share equivalents ​ ​ 8,738,097 ​ ​ 7,923,637 ​

Subsequent events

Subsequent events	3 Months Ended
	Mar. 31, 2024
Subsequent events
Subsequent events	13 ​ None.

Summary of significant accoun_2

Summary of significant accounting policies (Policies)	3 Months Ended
	Mar. 31, 2024
Summary of significant accounting policies
Basis of preparation	2.1 Basis 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 Update (“ASU”) of the Financial Accounting Standards Board (“FASB”). ​ 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.
Unaudited interim financial information	2.2 Unaudited 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 months ended March 31, 2024, are not necessarily indicative of the results to be expected for the full year ending December 31, 2024, 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
Use of estimates	2.3 Use 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 assets and liabilities at the date of the financial statements and reported amounts of revenues and expenses during the reporting period. Actual results could differ from those estimates.
Accounting policies	2.4 Accounting 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, 2023, and the notes thereto, which are included in the Annual Report ​
Recent accounting pronouncements	2.5 Recent accounting pronouncements ​ There have been no new accounting pronouncements or changes to accounting pronouncements during the three months ended March 31, 2024, as compared to the recent accounting pronouncements described in Note 2.3.27 of the Annual Report

Investment securities (Tables)

Investment securities (Tables)	3 Months Ended
	Mar. 31, 2024
Investment securities
Schedule of investments in sovereign debt	​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ At March 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) ​ $ 312,621 ​ $ 12 ​ $ — ​ $ 312,633 Total ​ $ 312,621 ​ $ 12 ​ $ — ​ $ 312,633 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ At December 31, 2023 ​ ​ Amortized cost ​ Gross unrealized holding gains ​ Gross unrealized holding losses ​ Estimated fair value ​ ​ (in thousands) Current investments: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Government debt securities (held-to-maturity) ​ $ 376,532 ​ $ 139 ​ $ — ​ $ 376,671 Total ​ $ 376,532 ​ $ 139 ​ $ — ​ $ 376,671

Inventories, net (Tables)

Inventories, net (Tables)	3 Months Ended
	Mar. 31, 2024
Inventories, net
Schedule of inventories, net balances	​ ​ ​ ​ ​ ​ ​ ​ ​ ​ March 31, ​ December 31, ​ 2024 2023 ​ ​ (in thousands) Raw materials ​ $ 6,331 ​ $ 7,157 Work in progress ​ ​ 1,341 ​ ​ 4,109 Finished goods ​ ​ — ​ ​ 758 Inventories ​ $ 7,672 ​ $ 12,024

Fair value measurement (Tables)

Fair value measurement (Tables)	3 Months Ended
	Mar. 31, 2024
Fair value measurement
Schedule of assets and liabilities measured at fair value on recurring basis	​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ 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) ​ ​ At December 31, 2023 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Assets: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Cash and cash equivalents ​ $ 241,360 ​ $ — ​ $ — ​ $ 241,360 ​ Cash and cash equivalents Restricted cash ​ ​ 3,184 ​ ​ — ​ ​ — ​ ​ 3,184 ​ Other non-current assets Total assets ​ $ 244,544 ​ $ — ​ $ — ​ $ 244,544 ​ ​ Liabilities: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Contingent consideration ​ ​ — ​ ​ — ​ ​ 43,006 ​ ​ 43,006 ​ Contingent consideration Consideration for post-acquisition services ​ ​ — ​ ​ — ​ ​ 457 ​ ​ 457 ​ Other non-current liabilities Total liabilities ​ $ — ​ $ — ​ $ 43,463 ​ $ 43,463 ​ ​ At March 31, 2024 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Assets: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Cash and cash equivalents ​ $ 243,062 ​ $ — ​ $ — ​ $ 243,062 ​ Cash and cash equivalents Restricted cash ​ ​ 3,167 ​ ​ — ​ ​ — ​ ​ 3,167 ​ Other non-current assets Total assets ​ $ 246,229 ​ $ — ​ $ — ​ $ 246,229 ​ ​ Liabilities: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Contingent consideration ​ ​ — ​ ​ — ​ ​ 42,212 ​ ​ 42,212 ​ Contingent consideration Consideration for post-acquisition services ​ ​ — ​ ​ — ​ ​ 498 ​ ​ 498 ​ Other non-current liabilities Total liabilities ​ $ — ​ $ — ​ $ 42,710 ​ $ 42,710 ​ ​
Schedule of changes in fair value of contingent consideration	​ ​ ​ ​ ​ Amount of ​ contingent ​ consideration ​ ​ 2024 ​ (in thousands) Balance at December 31, 2023 $ 43,006 Change in fair value (presented within research and development expenses) ​ 165 Currency translation effects ​ (959) Balance at March 31, 2024 $ 42,212

Accrued expenses and other cu_2

Accrued expenses and other current liabilities (Tables)	3 Months Ended
	Mar. 31, 2024
Accrued expenses and other current liabilities
Schedule of accrued expenses and other current liabilities	​ ​ ​ ​ ​ ​ ​ ​ ​ ​ March 31, ​ December 31, ​ 2024 2023 ​ ​ (in thousands) Personnel related accruals and liabilities ​ $ 9,572 ​ $ 16,263 Accruals for goods received from and services provided by vendors-not yet billed ​ ​ 11,881 ​ ​ 12,834 Liability owed to the Purchaser pursuant to the Royalty Financing Agreement ​ ​ 1,205 ​ ​ 1,437 Total ​ $ 22,658 ​ $ 30,534 ​

Royalty financing agreement (Ta

Royalty financing agreement (Tables)	3 Months Ended
	Mar. 31, 2024
Royalty Financing Agreement
Schedule of movement in the liability related to royalty financing agreement	​ ​ ​ ​ ​ ​ ​ ​ Amount of liability ​ ​ ​ (in thousands) Balance as of December 31, 2023 (includes $1.4 million presented as "Accrued expenses and other current liabilities) ​ $ 395,678 Royalty payments to Purchaser ​ ​ (1,437) Liability owed to the Purchaser (presented as "Accrued expense and other current liabilities") ​ ​ (1,205) Interest expense for the period ​ ​ 12,362 Liability related to the royalty financing agreement ​ $ 405,398 ​

Share-based compensation (Table

Share-based compensation (Tables)	3 Months Ended
	Mar. 31, 2024
Share-based compensation
Schedule of share-based compensation expense by classification included in consolidated statements of operations and comprehensive loss	​ ​ ​ ​ ​ ​ ​ ​ ​ Three months ended March 31, ​ ​ 2024 ​ 2023 ​ ​ (in thousands) Cost of manufacturing services revenue ​ $ 345 ​ $ 24 Research and development ​ ​ 3,425 ​ ​ 4,305 Selling, general and administrative ​ ​ 3,421 ​ ​ 3,732 Total ​ $ 7,191 ​ $ 8,061
Schedule of share-based compensation expense	​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three months ended March 31, ​ ​ 2024 ​ 2023 ​ ​ (in thousands) Award type/ESPP ​ ​ ​ ​ ​ ​ Share options ​ $ 2,913 ​ $ 3,274 Restricted share units ​ ​ 4,370 ​ ​ 4,630 Performance share units ​ ​ (99) ​ ​ 150 Employee share purchase plan ​ ​ 7 ​ ​ 7 Total ​ $ 7,191 ​ $ 8,061
Schedule of unrecognized compensation cost related to unvested awards	​ ​ ​ ​ ​ ​ ​ ​ Unrecognized Weighted average ​ share-based remaining ​ ​ compensation ​ period for ​ ​ expense ​ recognition ​ ​ (in thousands) ​ (in years) Award type ​ ​ ​ ​ ​ Share options ​ $ 20,743 ​ 2.56 Restricted share units ​ ​ 27,981 ​ 1.99 Performance share units ​ ​ 80 ​ 0.61 Total ​ $ 48,804 ​ 2.23
Schedule of weighted-average assumptions for fair value of option issued	​ ​ ​ ​ ​ ​ ​ ​ Three months ended March 31, Assumptions 2024 2023 Expected volatility ​ 70% ​ 70% Expected terms ​ 10 years ​ 10 years Risk free interest rate ​ 4.32% ​ 4.10% Expected dividend yield ​ 0% ​ 0%
Summary of RSUs activity	​ ​ ​ ​ ​ ​ ​ ​ ​ RSUs ​ ​ Weighted average ​ ​ Number of ​ grant-date fair ​ ​ ordinary shares ​ value Non-vested at December 31, 2023 ​ 2,264,369 ​ $ 18.07 Granted ​ 1,160,800 ​ $ 5.59 Vested ​ (659,240) ​ $ 20.30 Forfeited ​ (58,401) ​ $ 17.39 Non-vested at March 31, 2024 ​ 2,707,528 ​ $ 12.19 ​ ​ ​ ​ ​ ​ Total weighted average grant date fair value of RSUs granted during the period (in $ millions) ​ ​ ​ $ 6.5
Summary of PSUs activity	​ ​ ​ ​ ​ ​ ​ ​ ​ PSUs ​ ​ Weighted average ​ ​ Number of ​ grant-date fair ​ ​ ordinary shares ​ value Non-vested at December 31, 2023 ​ 222,550 ​ $ 28.09 Forfeited ​ (17,030) ​ $ 27.12 Non-vested at March 31, 2024 ​ 205,520 ​ $ 27.99
2014 Plan
Share-based compensation
Summary of option activity	​ ​ ​ ​ ​ ​ ​ ​ ​ Options ​ ​ Number of ​ Weighted average ​ ordinary shares exercise price ​ ​ ​ ​ ​ ​ Outstanding at December 31, 2023 ​ 4,974,030 ​ ​ 23.25 Granted ​ 930,900 ​ $ 5.59 Forfeited ​ (71,437) ​ $ 21.26 Expired ​ (11,584) ​ $ 37.61 Outstanding at March 31, 2024 ​ 5,821,909 ​ $ 20.42 Thereof, fully vested, and exercisable on March 31, 2024 ​ 3,054,242 ​ $ 25.70 Thereof, outstanding and expected to vest after March 31, 2024 ​ 2,767,667 ​ $ 14.60 Outstanding and expected to vest after December 31, 2023 ​ 2,098,557 ​ $ 23.38 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Total weighted average grant date fair value of options issued during the period (in $ millions) ​ ​ ​ $ 3.0 ​

Basic and diluted earnings pe_2

Basic and diluted earnings per share (Tables)	3 Months Ended
	Mar. 31, 2024
Basic and diluted earnings per share
Schedule of potential dilutive common shares	​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three months ended March 31, ​ ​ 2024 ​ 2023 Anti-dilutive ordinary share equivalents ​ ​ ​ ​ ​ ​ Stock options under 2014 Plans and previous plan ​ ​ 5,821,909 ​ ​ 5,150,690 Non-vested RSUs and PSUs ​ ​ 2,913,048 ​ ​ 2,772,146 ESPP ​ ​ 3,140 ​ ​ 801 Total anti-dilutive ordinary share equivalents ​ ​ 8,738,097 ​ ​ 7,923,637 ​

CSL Behring collaboration (Deta

CSL Behring collaboration (Details) - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2024	Mar. 31, 2023	Dec. 31, 2023
Collaboration arrangements
Revenue	$ 8,485	$ 5,325
CLS
Collaboration arrangements
Accounts receivable	10,600		$ 4,000
License revenues
Collaboration arrangements
Revenue	$ 1,202	$ 0

Investment securities (Details)

Investment securities (Details) - USD ($) $ in Thousands	Mar. 31, 2024	Dec. 31, 2023
Current investments:
Amortized cost	$ 312,621	$ 376,532
Gross unrealized holding gains	12	139
Estimated fair value	312,633	376,671
Government debt securities (held-to-maturity)
Current investments:
Amortized cost	312,621	376,532
Gross unrealized holding gains	12	139
Estimated fair value	$ 312,633	$ 376,671

Inventories, net (Details)

Inventories, net (Details) - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2024	Mar. 31, 2023	Dec. 31, 2023
Inventories, net
Raw materials	$ 6,331		$ 7,157
Work in progress	1,341		4,109
Finished goods			758
Inventories	7,672		12,024
Inventory write downs	1,800	$ 0
Allowance for inventory	$ 2,700		$ 1,600

Fair value measurement - Assets

Fair value measurement - Assets and liabilities measured on a recurring basis (Details) - USD ($) $ in Thousands	Mar. 31, 2024	Dec. 31, 2023	Mar. 31, 2023
Assets:
Restricted cash	$ 3,167		$ 3,173
Recurring Fair Value Measurements
Assets:
Cash and cash equivalents	243,062	$ 241,360
Restricted cash	3,167	3,184
Total assets	246,229	244,544
Liabilities:
Contingent consideration	42,212	43,006
Consideration for post-acquisition services	498	457
Total liabilities	42,710	43,463
Recurring Fair Value Measurements | Fair value hierarchy Level 1
Assets:
Cash and cash equivalents	243,062	241,360
Restricted cash	3,167	3,184
Total assets	246,229	244,544
Recurring Fair Value Measurements | Fair value hierarchy Level 3
Liabilities:
Contingent consideration	42,212	43,006
Consideration for post-acquisition services	498	457
Total liabilities	$ 42,710	$ 43,463

Fair value measurement - Contin

Fair value measurement - Contingent Consideration (Details) $ in Thousands, € in Millions	3 Months Ended			12 Months Ended
	Mar. 31, 2024 USD ($)	Mar. 31, 2024 EUR (€)	Mar. 31, 2023 USD ($)	Dec. 31, 2023 USD ($)	Mar. 31, 2024 EUR (€)
Fair Value, Liabilities Measured on Recurring Basis, Unobservable Input Reconciliation [Line Items]
Contingent consideration, net of current portion	$ 14,625			$ 14,795
Changes in fair value of contingent consideration	165		$ 975
Contingent consideration	42,200			43,000
Current portion of contingent consideration	$ 27,587			28,211
One hundred percent likelihood of AMT-260 advancing into clinical development
Fair Value, Liabilities Measured on Recurring Basis, Unobservable Input Reconciliation [Line Items]
Percentage of increase in Probability of Success	100%	100%
Changes in fair value of contingent consideration	$ 74,800
uniQure France [S.A.S]
Fair Value, Liabilities Measured on Recurring Basis, Unobservable Input Reconciliation [Line Items]
Contingent consideration, net of current portion	$ 42,212			$ 43,006
Percentage of increase in Probability of Success	100%	100%
Milestone payment achieving due to increase In Probability Of Success In Clinical Development	$ 32,400	€ 30
Changes in fair value of contingent consideration	165
uniQure France [S.A.S] | Maximum
Fair Value, Liabilities Measured on Recurring Basis, Unobservable Input Reconciliation [Line Items]
Contingent consideration payable upon achievement of contractually defined milestones	$ 193,000				€ 178.8
Contingent consideration discount rate percentage	15.60%	15.60%		15.60%
uniQure France [S.A.S] | Minimum
Fair Value, Liabilities Measured on Recurring Basis, Unobservable Input Reconciliation [Line Items]
Contingent consideration discount rate percentage	14.80%	14.80%		15.30%

Fair value measurement - Change

Fair value measurement - Changes in fair value of contingent consideration (Details) - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2024	Mar. 31, 2023
Fair Value, Liabilities Measured on Recurring Basis, Unobservable Input Reconciliation [Line Items]
Beginning Balance	$ 14,795
Change in fair value (presented within research and development expenses)	165	$ 975
Ending Balance	14,625
uniQure France [S.A.S]
Fair Value, Liabilities Measured on Recurring Basis, Unobservable Input Reconciliation [Line Items]
Beginning Balance	43,006
Change in fair value (presented within research and development expenses)	165
Currency translation effects	(959)
Ending Balance	$ 42,212

Accrued expenses and other cu_3

Accrued expenses and other current liabilities (Details) - USD ($) $ in Thousands	Mar. 31, 2024	Dec. 31, 2023
Accrued expenses and other current liabilities
Personnel related accruals and liabilities	$ 9,572	$ 16,263
Accruals for goods received from and services provided by vendors-not yet billed	11,881	12,834
Liability owed to the Purchaser pursuant to the Royalty Financing Agreement	1,205	1,437
Total	$ 22,658	$ 30,534

Long-term debt (Details)

Long-term debt (Details) - USD ($) $ in Millions		3 Months Ended
	Apr. 01, 2024	Mar. 31, 2024	Mar. 31, 2023	Jan. 05, 2027	Dec. 01, 2025	Dec. 31, 2023
Long-term Debt
Investment, Variable Interest Rate, Type [Extensible Enumeration]		Prime Rate
2023 Amended Facility
Long-term Debt
Outstanding debt		$ 100
Interest rate (as a percent)		7.95%
Amortized cost net of discount and debt issuance costs		$ 103.3				$ 102.9
Foreign currency loss		2.3
Foreign currency gain			$ 0.7
Interest expense recorded		$ 3.7	$ 3.6
2023 Amended Facility | Forecast
Long-term Debt
Back-end fees				$ 1.3	$ 4.9
2023 Amended Facility | Prime Rate
Long-term Debt
Variable interest rate basis		7.95%
Discount rate (as a percent)		3.25%
Venture debt loan facility | 2023 Amended Facility
Long-term Debt
Minimum cash and cash equivalents in U.S. bank accounts		65%	100%
Assets pledged to secure facilities by directly or indirectly		$ 770.1
Assets not being pledged to secure facilities by directly or indirectly		6.8
Venture debt loan facility | 2023 Amended Facility | uniQure France
Long-term Debt
Assets not being pledged to secure facilities by directly or indirectly		$ 87.7
Subsequent events. | Venture debt loan facility | 2023 Amended Facility
Long-term Debt
Minimum cash and cash equivalents in U.S. bank accounts	30%

Royalty financing agreement (De

Royalty financing agreement (Details) $ in Thousands		3 Months Ended
	May 12, 2023 USD ($)	Mar. 31, 2024 USD ($)	Mar. 31, 2023 USD ($)	Dec. 31, 2038	Jun. 30, 2032 USD ($)
Collaboration arrangements
Royalty financing agreement interest expense		$ 12,362
Interest Expense		16,097	$ 3,562
CLS
Collaboration arrangements
Maximum rights to all other royalties, as well as contractual milestones retained		1,300,000
Royalty financing agreement
Collaboration arrangements
Upfront payment received	$ 375,000
Additional milestone payment	$ 25,000
Ratio of upfront payment	1.85
Upfront payment payable to purchaser	$ 693,800	693,800
Royalty financing agreement interest expense		12,362
Net proceeds from the Royalty Financing Agreement		370,100
Payment of debt issuance costs		4,900
Interest Expense		$ 12,400	$ 0
Royalty financing agreement | Minimum
Collaboration arrangements
Effective interest rate (as a percent)		12%
Interest Expense		$ 12,100
Royalty financing agreement | Maximum
Collaboration arrangements
Effective interest rate (as a percent)		13.50%
Interest Expense		$ 13,700
Royalty financing agreement | First Hard Cap Date
Collaboration arrangements
Ratio of upfront payment					1.85
Upfront payment payable to purchaser					$ 25,000
Royalty financing agreement interest expense		323,700
Royalty financing agreement | If paid through December 31, 2038
Collaboration arrangements
Ratio of upfront payment				2.25
Royalty financing agreement | If 2024 net sales do not exceed a pre-specified threshold
Collaboration arrangements
Upfront payment payable to purchaser		$ 25,000

Royalty financing agreement - M

Royalty financing agreement - Movement in the liability (Details) - USD ($) $ in Thousands	3 Months Ended	12 Months Ended
	Mar. 31, 2024	Dec. 31, 2023
Collaboration arrangements
Liability owed to the Purchaser (presented as "Accrued expense and other current liabilities")	$ (1,205)	$ (1,437)
Interest expense for the period	12,362
Liability related to the royalty financing agreement	405,398	394,241
Royalty financing agreement
Collaboration arrangements
Balance as of December 31, 2023 (includes $1.4 million presented as "Accrued expenses and other current liabilities)		$ 395,678
Royalty payments to Purchaser	(1,437)
Liability owed to the Purchaser (presented as "Accrued expense and other current liabilities")	(1,205)
Interest expense for the period	12,362
Liability related to the royalty financing agreement	$ 405,398

Share-based compensation - Summ

Share-based compensation - Summary of share-based compensation expense and unrecognized costs (Details) - USD ($) $ in Thousands	1 Months Ended	3 Months Ended
	Jun. 30, 2018	Mar. 31, 2024	Mar. 31, 2023
Share-based compensation
Authorized shares		14
2014 Plan
Share-based compensation
Authorized shares		14
Share-based compensation expense		$ 7,191	$ 8,061
Unrecognized compensation costs		$ 48,804
Weighted-average remaining period for recognition (in years)		2 years 2 months 23 days
2014 Plans and ESPP
Share-based compensation
Share-based compensation expense		$ 7,191	8,061
Research and development expenses | 2014 Plans and ESPP
Share-based compensation
Share-based compensation expense		3,425	4,305
Selling, general and administrative expense | 2014 Plans and ESPP
Share-based compensation
Share-based compensation expense		3,421	3,732
Cost of manufacturing services revenues | 2014 Plans and ESPP
Share-based compensation
Share-based compensation expense		345	24
Share options | 2014 Plan
Share-based compensation
Unrecognized compensation costs		$ 20,743
Weighted-average remaining period for recognition (in years)		2 years 6 months 21 days
Share options | 2014 Plans and ESPP
Share-based compensation
Share-based compensation expense		$ 2,913	3,274
Restricted share units ("RSUs") | 2014 Plan
Share-based compensation
Unrecognized compensation costs		$ 27,981
Weighted-average remaining period for recognition (in years)		1 year 11 months 26 days
Restricted share units ("RSUs") | 2014 Plans and ESPP
Share-based compensation
Share-based compensation expense		$ 4,370	4,630
Performance Share Awards | 2014 Plan
Share-based compensation
Unrecognized compensation costs		$ 80
Weighted-average remaining period for recognition (in years)		7 months 9 days
Performance Share Awards | 2014 Plans and ESPP
Share-based compensation
Share-based compensation expense		$ (99)	$ 150
Employee share purchase plan | Employee share purchase plan (ESPP)
Share-based compensation
Ordinary shares available for issue	150,000	96,862	113,565
Discounted rate for purchase of shares	85%
Employee share purchase plan | 2014 Plans and ESPP
Share-based compensation
Share-based compensation expense		$ 7	$ 7

Share-based compensation - Opti

Share-based compensation - Option activity and weighted-average assumptions (Details) - USD ($) $ / shares in Units, $ in Thousands	3 Months Ended		12 Months Ended
	Mar. 31, 2024	Mar. 31, 2023	Dec. 31, 2023
Weighted average exercise price
Total weighted average grant date fair value of options issued during the period (in $ millions)	$ 3,000
Proceeds from Issuance of Shares under Incentive and Share-based Compensation Plans, Including Stock Options		$ 131
The 2014 Plan [Member]
Weighted average exercise price
Vesting period (in years)	4 years
The 2014 Plan [Member] | Share options
Options
Outstanding at beginning of the period (in shares)	4,974,030
Granted (in shares)	930,900
Forfeited (in shares)	(71,437)
Expired (in shares)	(11,584)
Outstanding at end of the period (in shares)	5,821,909		4,974,030
Thereof, fully vested and exercisable at end of period (in shares)	3,054,242
Thereof, outstanding and expected to vest after at end of period (in shares)	2,767,667		2,098,557
Weighted average exercise price
Outstanding at beginning of the period (in dollars per share)	$ 23.25
Granted (in dollars per share)	5.59
Forfeited (in dollars per share)	21.26
Expired (in dollars per share)	37.61
Outstanding at end of period (in dollars per share)	20.42		$ 23.25
Thereof, fully vested and exercisable at end of period (in dollars per share)	25.70
Outstanding and expected to vest after at end of the period (in dollars per share)	$ 14.60		$ 23.38
Weighted-average assumptions used to estimate fair value of share options granted during year
Expected volatility (as a percent)	70%	70%
Expected terms (in years)	10 years	10 years
Risk free interest rate (as a percent)	4.32%	4.10%
Expected dividend (as a percent)	0%	0%
The 2014 Plan [Member] | Non-executive directors
Weighted average exercise price
Vesting period (in years)	1 year
The 2014 Plan [Member] | One year from grant date
Weighted average exercise price
Vesting percentage per year	25%
Vesting period (in years)	1 year

Share-based compensation - RSU

Share-based compensation - RSU activity (Details) - The 2014 Plan [Member] $ / shares in Units, $ in Millions	3 Months Ended
	Mar. 31, 2024 USD ($) $ / shares shares
Other disclosure
Vesting period (in years)	4 years
Non-executive directors
Other disclosure
Vesting period (in years)	1 year
Restricted share units ("RSUs")
Number of shares
Non-vested at beginning of period (in shares) | shares	2,264,369
Granted (in shares) | shares	1,160,800
Vested (in shares) | shares	(659,240)
Forfeited (in shares) | shares	(58,401)
Non-vested at end of period (in shares) | shares	2,707,528
Weighted average grant-date fair value
Non-vested at beginning of period (in dollars per share) | $ / shares	$ 18.07
Granted (in dollars per share) | $ / shares	5.59
Vested (in dollars per share) | $ / shares	20.30
Forfeited (in dollars per share) | $ / shares	17.39
Non-vested at end of period (in dollars per share) | $ / shares	$ 12.19
Other disclosure
Total weighted average grant date fair value of RSUs granted during the period (in millions) | $	$ 6.5
Restricted share units ("RSUs") | Non-executive directors
Other disclosure
Vesting period (in years)	1 year
Minimum | Restricted share units ("RSUs")
Other disclosure
Vesting period (in years)	1 year
Maximum | Restricted share units ("RSUs")
Other disclosure
Vesting period (in years)	3 years

Share-based compensation - PSU

Share-based compensation - PSU activity (Details)	3 Months Ended
	Mar. 31, 2024 Milestone $ / shares shares	Dec. 31, 2023 Milestone
Other disclosure
Number of Milestones Achieved | Milestone	2
Number of Milestones Achievement Considered Probable | Milestone	2	2
Minimum
Other disclosure
Service period	1 year
Maximum
Other disclosure
Service period	3 years
Performance Share Awards | 2014 Plan
Number of shares
Non-vested at beginning of period (in shares) | shares	222,550
Forfeited (in shares) | shares	(17,030)
Non-vested at end of period (in shares) | shares	205,520
Weighted average grant-date fair value
Non-vested at beginning of period (in dollars per share) | $ / shares	$ 28.09
Forfeited (in dollars per share) | $ / shares	27.12
Non-vested at end of period (in dollars per share) | $ / shares	$ 27.99

Share-based compensation - Empl

Share-based compensation - Employee Share Purchase Plan (Details) - Employee share purchase plan (ESPP) - Employee Stock - shares	3 Months Ended
	Mar. 31, 2024	Mar. 31, 2023	Jun. 30, 2018
Share-based compensation
Number of shares issued	0	2,495
Ordinary shares available for issue	96,862	113,565	150,000

Income taxes - Effective Tax Ra

Income taxes - Effective Tax Rate (Details) - USD ($) $ in Millions	3 Months Ended
	Mar. 31, 2024	Mar. 31, 2023
Effective income tax rate	1%	(1.50%)
Enacted tax rate	25.80%
U.S
Deferred tax (expense) / benefit	$ (0.7)
United States and France
Deferred tax (expense) / benefit		$ 1.2

Basic and diluted earnings pe_3

Basic and diluted earnings per share (Details) - shares	3 Months Ended
	Mar. 31, 2024	Mar. 31, 2023
Basic and diluted earnings per share
Total anti-dilutive ordinary share equivalents	8,738,097	7,923,637
ESPP
Basic and diluted earnings per share
Total anti-dilutive ordinary share equivalents	3,140	801
Employee Stock Option
Basic and diluted earnings per share
Total anti-dilutive ordinary share equivalents	5,821,909	5,150,690
Non-vested RSUs and PSUs
Basic and diluted earnings per share
Total anti-dilutive ordinary share equivalents	2,913,048	2,772,146

Insider Trading Arrangements

Insider Trading Arrangements	3 Months Ended
	Mar. 31, 2024
Trading Arrangements, by Individual
Rule 10b5-1 Arrangement Adopted	false
Non-Rule 10b5-1 Arrangement Adopted	false
Rule 10b5-1 Arrangement Terminated	false
Non-Rule 10b5-1 Arrangement Terminated	false