uniQure (QURE) 10-Q7 Nov 232023 Q3 Quarterly reportFinancial data

Company Profile

Document and Entity Information

Document and Entity Information - shares	9 Months Ended
Document and Entity Information - shares	Sep. 30, 2023	Nov. 02, 2023
Document and Entity Information
Document Type	10-Q
Document Quarterly Report	true
Document Period End Date	Sep. 30, 2023
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	Large Accelerated Filer
Entity Small Business	false
Entity Emerging Growth Company	false
Entity Shell Company	false
Entity Common Stock, Shares Outstanding		47,811,479
Entity Central Index Key	0001590560
Current Fiscal Year End Date	--12-31
Document Fiscal Year Focus	2023
Document Fiscal Period Focus	Q3
Amendment Flag	false

UNAUDITED CONSOLIDATED BALANCE

UNAUDITED CONSOLIDATED BALANCE SHEETS - USD ($) $ in Thousands	Sep. 30, 2023	Dec. 31, 2022
Current assets
Cash and cash equivalents	$ 229,484	$ 228,012
Current investment securities	429,428	124,831
Accounts receivable and contract asset	1,644	102,376
Inventories	15,654	6,924
Prepaid expenses	14,884	11,817
Other current assets and receivables	2,532	2,814
Total current assets	693,626	476,774
Non-current assets
Property, plant and equipment, net of accumulated depreciation of $51.6 million as of September 30, 2023 and $44.1 million as of December 31, 2022	45,946	50,532
Non-current investment securities		39,984
Operating lease right-of-use assets	30,360	32,726
Intangible assets, net, including in-process research and development asset of $56.6 million as of September 30, 2023 and $57.3 million as of December 31, 2022	57,976	58,778
Goodwill	25,273	25,581
Deferred tax assets, net	12,351	14,528
Other non-current assets	6,018	6,061
Total non-current assets	177,924	228,190
Total assets	871,550	704,964
Current liabilities
Accounts payable	5,584	10,984
Accrued expenses and other current liabilities	28,427	30,571
Current portion of contingent consideration	26,708	25,982
Current portion of operating lease liabilities	7,888	8,382
Total current liabilities	68,607	75,919
Non-current liabilities
Long-term debt	101,431	102,791
Liability from royalty financing agreement	383,711
Operating lease liabilities, net of current portion	28,977	31,719
Contingent consideration, net of current portion	14,030	9,334
Deferred tax liability, net	4,917	8,257
Other non-current liabilities	1,093	935
Total non-current liabilities	534,159	153,036
Total liabilities	602,766	228,955
Commitments and contingencies
Shareholders' equity
Ordinary shares, €0.05 par value: 80,000,000 shares authorized as of September 30, 2023 and December 31, 2022 and 47,810,291 and 46,968,032 ordinary shares issued and outstanding as of September 30, 2023 and December 31, 2022, respectively	2,883	2,838
Additional paid-in-capital	1,141,662	1,113,393
Accumulated other comprehensive loss	(58,558)	(58,291)
Accumulated deficit	(817,203)	(581,931)
Total shareholders' equity	268,784	476,009
Total liabilities and shareholders' equity	$ 871,550	$ 704,964

UNAUDITED CONSOLIDATED BALANC_2

UNAUDITED CONSOLIDATED BALANCE SHEETS (Parenthetical) $ in Thousands	Sep. 30, 2023 USD ($) shares	Sep. 30, 2023 € / shares	Dec. 31, 2022 USD ($) shares	Dec. 31, 2022 € / shares
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	47,810,291		46,968,032
Ordinary shares, outstanding \| shares	47,810,291		46,968,032
Accumulated depreciation \| $	$ 51,600		$ 44,100
Intangible assets, net \| $	57,976		58,778
In-process research & development
Intangible assets, net \| $	$ 56,600		$ 57,300

UNAUDITED CONSOLIDATED STATEMEN

UNAUDITED CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2023	Sep. 30, 2022	Sep. 30, 2023	Sep. 30, 2022
Total revenues	$ 1,407	$ 1,449	$ 9,154	$ 3,738
Operating expenses:
Cost of contract manufacturing revenues	(1,006)	(861)	(4,793)	(1,693)
Research and development expenses	(65,400)	(48,068)	(172,245)	(139,263)
Selling, general and administrative expenses	(18,074)	(13,324)	(57,103)	(36,802)
Total operating expenses	(84,480)	(62,253)	(234,141)	(177,758)
Other income	1,424	1,485	4,537	4,981
Other expense	(228)	(199)	(673)	(621)
Loss from operations	(81,877)	(59,518)	(221,123)	(169,660)
Interest income	7,495	39	12,393	117
Interest expense	(15,444)	(3,069)	(25,846)	(8,279)
Foreign currency gains / (losses), net	186	14,362	(1,809)	42,328
Other non-operating gains, net				635
Loss before income tax benefit	(89,640)	(48,186)	(236,385)	(134,859)
Income tax benefit	69	329	1,113	1,263
Net loss	(89,571)	(47,857)	(235,272)	(133,596)
Other comprehensive loss:
Foreign currency translation adjustments	(6,118)	(25,370)	(267)	(64,144)
Total comprehensive loss	$ (95,689)	$ (73,227)	$ (235,539)	$ (197,740)
Earnings per ordinary share - basic
Basic net loss per ordinary share	$ (1.88)	$ (1.02)	$ (4.94)	$ (2.86)
Earnings per ordinary share - diluted
Diluted net loss per ordinary share	$ (1.88)	$ (1.02)	$ (4.94)	$ (2.86)
Weighted average shares - basic	47,770,101	46,772,430	47,619,875	46,680,667
Weighted average shares - diluted	47,770,101	46,772,430	47,619,875	46,680,667
License revenues
Total revenues	$ 497	$ 0	$ 1,290	$ 0
Contract manufacturing revenues
Total revenues	349		6,596
Collaboration revenues
Total revenues	$ 561	$ 1,449	$ 1,268	$ 3,738

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, 2021	$ 2,802	$ 1,076,972	$ (28,856)	$ (455,142)	$ 595,776
Beginning balance (in shares) at Dec. 31, 2021	46,298,635
Increase (decrease) in shareholders' equity
Loss for the period				(133,596)	(133,596)
Other comprehensive loss			(64,144)		(64,144)
Exercise of share options	$ 6	707			713
Exercise of share options (in shares)	119,884
Restricted and performance share units distributed during the period	$ 21	(21)
Restricted and performance share units distributed during the period (in shares)	387,285
Share-based compensation expense		22,290			22,290
Issuance of ordinary shares relating to employee stock purchase plan	$ 1	130			131
Issuance of ordinary shares relating to employee stock purchase plan (in shares)	9,305
Ending balance at Sep. 30, 2022	$ 2,830	1,100,078	(93,000)	(588,738)	421,170
Ending balance (in shares) at Sep. 30, 2022	46,815,109
Beginning balance at Jun. 30, 2022	$ 2,823	1,092,176	(67,630)	(540,881)	486,488
Beginning balance (in shares) at Jun. 30, 2022	46,684,583
Increase (decrease) in shareholders' equity
Loss for the period				(47,857)	(47,857)
Other comprehensive loss			(25,370)		(25,370)
Exercise of share options	$ 2	258			260
Exercise of share options (in shares)	47,290
Restricted and performance share units distributed during the period	$ 4	(4)
Restricted and performance share units distributed during the period (in shares)	79,821
Share-based compensation expense		7,608			7,608
Issuance of ordinary shares relating to employee stock purchase plan	$ 1	40			41
Issuance of ordinary shares relating to employee stock purchase plan (in shares)	3,415
Ending balance at Sep. 30, 2022	$ 2,830	1,100,078	(93,000)	(588,738)	421,170
Ending balance (in shares) at Sep. 30, 2022	46,815,109
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				46,968,032
Increase (decrease) in shareholders' equity
Loss for the period				(235,272)	$ (235,272)
Other comprehensive loss			(267)		(267)
Exercise of share options	$ 1	122			123
Exercise of share options (in shares)	12,882
Restricted and performance share units distributed during the period	$ 43	(43)
Restricted and performance share units distributed during the period (in shares)	817,107
Share-based compensation expense		28,052			28,052
Issuance of ordinary shares relating to employee stock purchase plan	$ 1	138			139
Issuance of ordinary shares relating to employee stock purchase plan (in shares)	12,270
Ending balance at Sep. 30, 2023	$ 2,883	1,141,662	(58,558)	(817,203)	$ 268,784
Ending balance (in shares) at Sep. 30, 2023	47,810,291				47,810,291
Beginning balance at Jun. 30, 2023	$ 2,877	1,130,515	(52,440)	(727,632)	$ 353,320
Beginning balance (in shares) at Jun. 30, 2023	47,702,331
Increase (decrease) in shareholders' equity
Loss for the period				(89,571)	(89,571)
Other comprehensive loss			(6,118)		(6,118)
Exercise of share options		2			2
Exercise of share options (in shares)	400
Restricted and performance share units distributed during the period	$ 5	(5)
Restricted and performance share units distributed during the period (in shares)	100,296
Share-based compensation expense		11,097			11,097
Issuance of ordinary shares relating to employee stock purchase plan	$ 1	53			54
Issuance of ordinary shares relating to employee stock purchase plan (in shares)	7,264
Ending balance at Sep. 30, 2023	$ 2,883	$ 1,141,662	$ (58,558)	$ (817,203)	$ 268,784
Ending balance (in shares) at Sep. 30, 2023	47,810,291				47,810,291

UNAUDITED CONSOLIDATED STATEM_3

UNAUDITED CONSOLIDATED STATEMENTS OF CASH FLOWS - USD ($) $ in Thousands	9 Months Ended
	Sep. 30, 2023	Sep. 30, 2022
Cash flows from operating activities
Net loss	$ (235,272)	$ (133,596)
Adjustments to reconcile net loss to net cash used in operating activities:
Depreciation and amortization expense	7,712	6,206
Amortization of premium/discount on investment securities	(6,529)
Share-based compensation expense	28,052	22,290
Royalty financing agreement interest expense	14,940
Deferred tax income	(1,113)	(1,263)
Changes in fair value of contingent consideration and derivative financial instrument, net	15,441	6,875
Unrealized foreign exchange losses / (gains), net	3,119	(36,390)
Other items, net	2,146	(581)
Changes in operating assets and liabilities:
Accounts receivable and contract asset, prepaid expenses, and other current assets and receivables	96,551	41,992
Inventories	(8,730)	(4,075)
Accounts payable	(4,784)	4,529
Accrued expenses, other liabilities, and operating leases	(6,007)	3,633
Contingent consideration milestone payment	(1,914)
Net cash used in operating activities	(96,388)	(90,380)
Cash flows from investing activities
Investment in investment securities	(366,439)
Proceeds on maturity of investment securities	106,307
Purchases of property, plant, and equipment	(5,116)	(12,622)
Acquisition of uniQure France SAS (formerly Corlieve Therapeutics SAS), net of cash acquired		(1,900)
Net cash used in investing activities	(265,248)	(14,522)
Cash flows from financing activities
Proceeds from royalty financing agreement	374,350
Payment of debt issuance costs	(4,288)
Proceeds from issuance of ordinary shares related to employee stock option and purchase plans	262	844
Contingent consideration milestone payment	(7,649)
Net cash generated from financing activities	362,675	844
Currency effect on cash, cash equivalents and restricted cash	424	(11,876)
Net increase / (decrease) in cash, cash equivalents and restricted cash	1,463	(115,934)
Cash, cash equivalents and restricted cash at beginning of period	231,173	559,353
Cash, cash equivalents and restricted cash at the end of period	232,636	443,419
Cash and cash equivalents	229,484	440,313
Restricted cash related to leasehold and other deposits	3,152	3,106
Total cash, cash equivalents and restricted cash	232,636	443,419
Supplemental cash flow disclosures:
Cash paid for interest	(12,996)	(6,410)
Non-cash decrease in accounts payables and accrued expenses and other current liabilities related to purchases of property, plant, and equipment	$ (995)	$ (1,766)

General business information

General business information	9 Months Ended
General business information	Sep. 30, 2023
General business information
General business information	1 uniQure N.V. (the “Company”) was incorporated on January 9, 2012, initially as a private company with limited liability ( besloten vennootschap met beperkte aansprakelijkheid naamloze vennootschap The Company is registered in the trade register of the 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	9 Months Ended
Summary of significant accounting policies	Sep. 30, 2023
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 of America (“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 and nine months ended September 30, 2023, are not necessarily indicative of the results to be expected for the full year ending December 31, 2023, 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 periods. 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, 2022, and the notes thereto, which are included in the Annual Report Royalty Financing Agreement In May 2023, uniQure biopharma B.V. (“uniQure biopharma”), a wholly-owned subsidiary of the Company entered into an agreement (the “Royalty Financing Agreement”) with HemB SPV (the “Purchaser”) to sell certain current and future royalties due to uniQure biopharma from CSL Behring LLC (“CSL Behring”) under the Commercialization and License Agreement (the “CSL Behring Agreement”) by and between uniQure biopharma and CSL Behring from the net sales of HEMGENIX®. Refer to Note 9 “Royalty Financing Agreement” ASC 470, Debt In accordance with topic ASC 835, Interest 2.5 Recent accounting pronouncements There have been no new accounting pronouncements or changes to accounting pronouncements during the nine months ended September 30, 2023, as compared to the recent accounting pronouncements described in Note 2.3.25 of the Annual Report

CSL Behring collaboration

CSL Behring collaboration	9 Months Ended
CSL Behring collaboration	Sep. 30, 2023
CSL Behring collaboration
CSL Behring collaboration	3 CSL Behring collaboration On June 24, 2020, uniQure biopharma B.V. entered into the CSL Behring Agreement with CSL Behring, pursuant to which CSL Behring received exclusive global rights to HEMGENIX® License revenue The Company recognized $0.5 million and $1.3 million of royalty revenue in each of the three and nine months ended September 30, 2023, compared to nil in the three and nine months ended September 30, 2022. Royalties on the sale of the HEMGENIX® are recorded once earned and are presented as license revenue. Accounts receivable and contract asset As of December 31, 2022, the Company recorded accounts receivable of $2.2 million from CSL Behring related to collaboration services as well as a contract asset of $100.0 million for a milestone due from CSL Behring following the first sale of , which was collected in July 2023. As of September 30, 2023, the Company had accounts receivable of $1.6 million from CSL Behring related to collaboration services, contract manufacturing revenue and royalty revenue.

Investment securities

Investment securities	9 Months Ended
Investment securities	Sep. 30, 2023
Investment securities
Investment securities	4 Investment securities The following tables summarize the Company’s investments in sovereign debt as of September 30, 2023 and December 31, 2022: At September 30, 2023 Amortized cost, as adjusted Gross unrealized holding gains Gross unrealized holding losses Estimated fair value (in thousands) Current investments: Government debt securities (held-to-maturity) $ 429,428 $ — $ (556) $ 428,872 Total $ 429,428 $ — $ (556) $ 428,872 At December 31, 2022 Amortized cost, as adjusted Gross unrealized holding gains Gross unrealized holding losses Estimated fair value (in thousands) Current investments: Government debt securities (held-to-maturity) $ 124,831 $ — $ (283) $ 124,548 Non-current investments: Government debt securities (held-to-maturity) 39,984 — (43) 39,941 Total $ 164,815 $ — $ (326) $ 164,489 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

Inventories	9 Months Ended
Inventories	Sep. 30, 2023
Inventories
Inventories	5 Inventories The following table summarizes the inventory balances as of September 30, 2023 and December 31, 2022: September 30, December 31, 2023 2022 (in thousands) Raw materials $ 7,426 $ 3,584 Work in progress 6,597 1,874 Finished goods 1,631 1,466 Inventories $ 15,654 $ 6,924

Fair value measurement

Fair value measurement	9 Months Ended
Fair value measurement	Sep. 30, 2023
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 September 30, 2023, and December 31, 2022: 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, 2022 Assets: Cash and cash equivalents $ 228,012 $ — $ — $ 228,012 Cash and cash equivalents Restricted cash 3,161 — — 3,161 Other non-current assets Total assets $ 231,173 $ — $ — $ 231,173 Liabilities: Contingent consideration — — 35,316 35,316 Contingent consideration Consideration for post-acquisition services — — 297 297 Other non-current liabilities Total liabilities $ — $ — $ 35,613 $ 35,613 At September 30, 2023 Assets: Cash and cash equivalents $ 229,484 $ — $ — $ 229,484 Cash and cash equivalents Restricted cash 3,152 — — 3,152 Other non-current assets Total assets $ 232,636 $ — $ — $ 232,636 Liabilities: Contingent consideration — — 40,738 40,738 Contingent consideration Consideration for post-acquisition services — — 478 478 Other non-current liabilities Total liabilities $ — $ — $ 41,216 $ 41,216 Contingent consideration The Company is required to pay up to EUR 178.8 million (or $189.1 million based on the foreign exchange rate on September 30, 2023) 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. In September 2023, a milestone payment of EUR 10.0 million ($10.6 million) was paid, of which EUR 8.9 million ($9.6 million) related to contingent consideration. The fair value of the contingent consideration as of September 30, 2023 was $40.7 million (December 31, 2022: $35.3 million) using discount rates of approximately 15.0% to 15.5% (December 31, 2022: 14.0% to 14.4%). Following the clearance of an Investigational New Drug (“IND”) application for AMT-260 in August 2023, the Company increased the probability of achieving a EUR 30.0 million ($31.7 million) milestone payment due to the first patient dosed in Phase I/II clinical trial from 66.0% to 100.0%. This also resulted in an increase of, the probability that AMT-260 may advance to late-stage development and commercialization. The Company recorded $14.2 million and $15.4 million in expenses related to the changes in the fair value of the contingent consideration in the three and nine months ended September 30, 2023, respectively, compared to $5.5 million and $7.5 million during the same period in 2022. If as of September 30, 2023 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 $72.0 million. If as of September 30, 2023 the Company had assumed that it would discontinue development of the AMT-260 program, then the contingent consideration would be released to income. Changes in fair value of the contingent consideration are recognized within research and development expenses in the consolidated statements of operations and comprehensive loss. The following table presents the changes in fair value of contingent consideration between December 31, 2022 and September 30, 2023: Amount of contingent consideration 2023 (in thousands) Balance at December 31, 2022 $ 35,316 Change in fair value (presented within research and development expenses) 15,441 Contingent consideration milestone payment (9,563) Currency translation effects (456) Balance at September 30, 2023 $ 40,738 As of September 30, 2023, the Company classified $26.7 million of the total contingent consideration of $40.7 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	9 Months Ended
Accrued expenses and other current liabilities	Sep. 30, 2023
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: September 30, December 31, 2023 2022 (in thousands) Accruals for goods received from and services provided by vendors-not yet billed $ 11,688 $ 11,120 Personnel related accruals and liabilities 16,244 17,201 Liability owed to the Purchaser pursuant to the Royalty Financing Agreement 495 — Accrued contract fulfillment costs and costs to obtain a contract — 2,250 Total $ 28,427 $ 30,571

Long-term debt

Long-term debt	9 Months Ended
Long-term debt	Sep. 30, 2023
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% or (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 $102.5 million as of September 30, 2023, compared to $103.8 million as of December 31, 2022, and is recorded net of discount and debt issuance costs. The foreign currency loss on the facility in the three and nine months ended September 30, 2023 was $3.0 million and $1.4 million, respectively, compared to a foreign currency loss of $6.6 million and $15.0 million during the same period in 2022. Interest expense associated with the 2023 Amended Facility during the three and nine months ended September 30, 2023 was $3.7 million and $10.9 million, respectively, compared to $3.0 million and $8.1 million during the same period in 2022. 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 $871.6 million, less $1.0 million of cash and cash equivalents and other current assets held by the Company, $85.0 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 September 30, 2023, the Company was in material compliance with all covenants and provisions.

Royalty Financing Agreement

Royalty Financing Agreement	9 Months Ended
Royalty Financing Agreement	Sep. 30, 2023
Royalty Financing Agreement
Royalty Financing Agreement	9 On May 12, 2023, the Company entered into the Royalty Financing Agreement with 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 certain thresholds, 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 the 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” 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 $14.6 million and $16.5 million of interest expense through the nine months ended September 30, 2023 ($11.5 million and $13.0 million, respectively, for the three months ended September 30, 2023) if it had used 12.0% or 13.5%, instead of the $14.9 million recorded in the nine months ended September 30, 2023 ($11.8 million recorded through the three months ended September 30, 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 closing of the transaction on June 5, 2023 and September 30, 2023: Amount of liability (in thousands) Gross proceeds from royalty financing agreement on June 5, 2023 $ 375,000 Debt issuance costs paid (4,938) Royalty payments to Purchaser (796) Liability owed to the Purchaser (presented as "Accrued expense and other current liabilities") (495) Interest expense for the period June 5, 2023 to September 30, 2023 14,940 Liability related to the royalty financing agreement $ 383,711

Share-based compensation

Share-based compensation	9 Months Ended
Share-based compensation	Sep. 30, 2023
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 12,601,471 . The 2014 Plan expires on January 9, 2024. 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 September 30, Nine months ended September 30, 2023 2022 2023 2022 (in thousands) (in thousands) Cost of manufacturing services revenue $ 315 $ 120 $ 477 $ 268 Research and development 5,638 3,730 14,675 12,090 Selling, general and administrative 5,144 3,758 12,900 9,932 Total $ 11,097 $ 7,608 $ 28,052 $ 22,290 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, 2023 2022 2023 2022 (in thousands) (in thousands) Award type/ESPP Share options $ 3,497 $ 3,448 $ 10,256 $ 10,141 Restricted share units 5,293 3,957 15,311 11,354 Performance share units 2,299 197 2,461 777 Employee share purchase plan 8 6 24 18 Total $ 11,097 $ 7,608 $ 28,052 $ 22,290 As of September 30, 2023, 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 $ 27,765 2.59 Restricted share units 35,763 2.03 Performance share units 556 1.20 Total $ 64,084 2.27 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. All share options must be exercised by the tenth anniversary of the initial grant date. The following tables summarize share option activity under the 2014 Plans for the nine months ended September 30, 2023: Options Number of Weighted average ordinary shares exercise price Outstanding at December 31, 2022 4,237,917 $ 26.13 Granted 1,575,430 $ 18.69 Forfeited (257,880) $ 22.82 Expired (131,026) $ 43.95 Exercised (12,882) $ 9.58 Outstanding at September 30, 2023 5,411,559 $ 23.73 Thereof, fully vested, and exercisable on September 30, 2023 2,826,806 $ 26.43 Thereof, outstanding and expected to vest after September 30, 2023 2,584,753 $ 20.76 Total weighted average grant date fair value of options issued during the period (in $ millions) $ 17.1 Proceeds from option sales during the period (in $ millions) $ 0.1 The fair value of each share 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, Assumptions 2023 2022 2023 2022 Expected volatility 70% 70% 70% 70% Expected terms 10 years 10 years 10 years 10 years Risk free interest rate 3.71% - 4.00% 4.16% 3.71% - 4.10% 2.12% - 4.16% Expected dividend yield 0% 0% 0% 0% RSUs The following table summarizes the RSU activity for the nine months ended September 30, 2023: RSUs Weighted average Number of grant-date fair ordinary shares value Non-vested at December 31, 2022 1,818,774 $ 20.46 Granted 1,614,925 $ 18.90 Vested (722,977) $ 22.57 Forfeited (224,402) $ 19.59 Non-vested at September 30, 2023 2,486,320 $ 18.91 Total weighted average grant date fair value of RSUs granted during the period (in $ millions) $ 30.5 RSUs vest over one PSUs The following table summarizes the PSU activity for the nine months ended September 30, 2023: PSUs Weighted average Number of grant-date fair ordinary shares value Non-vested at December 31, 2022 400,690 $ 28.82 Vested (94,510) $ 30.65 Forfeited (42,745) $ 28.04 Non-vested at September 30, 2023 263,435 $ 28.29 The Company granted ordinary shares to certain employees in September and December 2021 and at various dates during the year ended December 31, 2022 that will be earned upon the achievement of defined milestones. Such 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 September 30, 2023, two milestones had been achieved. Additionally, another milestone is considered probable as of September 30, 2023. The ESPP During the nine months ended September 30, 2023, 12,270 ordinary shares were issued under the ESPP compared to 9,305 during the same period in 2022. As of September 30, 2023, 103,790 ordinary shares remain available for issuance under the ESPP compared to a total of 117,997 as of September 30, 2022.

Income taxes

Income taxes	9 Months Ended
Income taxes	Sep. 30, 2023
Income taxes
Income taxes	11 Income taxes The Company recorded $0.1 million and $1.1 million deferred tax benefit in relation to its operations in the U.S. and France during the three and nine months ended September 30, 2023, respectively. The Company recorded $0.3 million and $1.3 million deferred tax benefit in relation to its operations in the U.S. and France during the three and nine months ended September 30, 2022, respectively. The effective income tax rate of (0.1%) and (0.5%) during the three and nine months ended September 30, 2023 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. The effective income tax rate during the three and nine months ended September 30, 2022 was (0.7%) and (0.9%), respectively, 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	9 Months Ended
Basic and diluted earnings per share	Sep. 30, 2023
Basic and diluted earnings per share
Basic and diluted earnings per share	12 Basic and diluted earnings per share The potentially dilutive ordinary shares are summarized below: Three months ended September 30, Nine months ended September 30, 2023 2022 2023 2022 Anti-dilutive ordinary share equivalents Stock options under 2014 Plans and previous plan 5,411,599 4,284,656 5,411,599 4,284,656 Non-vested RSUs and PSUs 2,749,755 2,328,151 2,749,755 2,328,151 ESPP 2,857 721 2,857 721 Total anti-dilutive ordinary share equivalents 8,164,211 6,613,528 8,164,211 6,613,528

Subsequent events

Subsequent events	9 Months Ended
Subsequent events	Sep. 30, 2023
Subsequent events
Subsequent events	13 On October 5, 2023, the Company announced a reorganization plan (the “Reorganization”) pursuant to which the Company’s global workforce will be reduced by approximately 20%. As part of the Reorganization, more than half of the Company’s research and technology projects will be discontinued and a research lab in Lexington, Massachusetts will be closed. The Company estimated that it will incur charges of approximately $2.3 million in connection with the reorganization related to cash expenditures for employee severance costs. The Company also anticipates that there could be costs associated with the lab to be closed in Lexington and associated fixed assets if the carrying value is determined to not be recoverable as of the cease-use date, which is expected to be later in the fourth quarter of 2023. The Company anticipates the costs associated with the reorganization will be incurred in the fourth quarter of 2023.

Summary of significant accoun_2

Summary of significant accounting policies (Policies)	9 Months Ended
Summary of significant accounting policies (Policies)	Sep. 30, 2023
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 of America (“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 and nine months ended September 30, 2023, are not necessarily indicative of the results to be expected for the full year ending December 31, 2023, 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 periods. 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, 2022, and the notes thereto, which are included in the Annual Report Royalty Financing Agreement In May 2023, uniQure biopharma B.V. (“uniQure biopharma”), a wholly-owned subsidiary of the Company entered into an agreement (the “Royalty Financing Agreement”) with HemB SPV (the “Purchaser”) to sell certain current and future royalties due to uniQure biopharma from CSL Behring LLC (“CSL Behring”) under the Commercialization and License Agreement (the “CSL Behring Agreement”) by and between uniQure biopharma and CSL Behring from the net sales of HEMGENIX®. Refer to Note 9 “Royalty Financing Agreement” ASC 470, Debt In accordance with topic ASC 835, Interest
Recent accounting pronouncements	2.5 Recent accounting pronouncements There have been no new accounting pronouncements or changes to accounting pronouncements during the nine months ended September 30, 2023, as compared to the recent accounting pronouncements described in Note 2.3.25 of the Annual Report

Investment securities (Tables)

Investment securities (Tables)	9 Months Ended
Investment securities (Tables)	Sep. 30, 2023
Investment securities
Schedule of investments into sovereign debt	At September 30, 2023 Amortized cost, as adjusted Gross unrealized holding gains Gross unrealized holding losses Estimated fair value (in thousands) Current investments: Government debt securities (held-to-maturity) $ 429,428 $ — $ (556) $ 428,872 Total $ 429,428 $ — $ (556) $ 428,872 At December 31, 2022 Amortized cost, as adjusted Gross unrealized holding gains Gross unrealized holding losses Estimated fair value (in thousands) Current investments: Government debt securities (held-to-maturity) $ 124,831 $ — $ (283) $ 124,548 Non-current investments: Government debt securities (held-to-maturity) 39,984 — (43) 39,941 Total $ 164,815 $ — $ (326) $ 164,489

Inventories (Tables)

Inventories (Tables)	9 Months Ended
Inventories (Tables)	Sep. 30, 2023
Inventories
Schedule of inventory balances	September 30, December 31, 2023 2022 (in thousands) Raw materials $ 7,426 $ 3,584 Work in progress 6,597 1,874 Finished goods 1,631 1,466 Inventories $ 15,654 $ 6,924

Fair value measurement (Tables)

Fair value measurement (Tables)	9 Months Ended
Fair value measurement (Tables)	Sep. 30, 2023
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, 2022 Assets: Cash and cash equivalents $ 228,012 $ — $ — $ 228,012 Cash and cash equivalents Restricted cash 3,161 — — 3,161 Other non-current assets Total assets $ 231,173 $ — $ — $ 231,173 Liabilities: Contingent consideration — — 35,316 35,316 Contingent consideration Consideration for post-acquisition services — — 297 297 Other non-current liabilities Total liabilities $ — $ — $ 35,613 $ 35,613 At September 30, 2023 Assets: Cash and cash equivalents $ 229,484 $ — $ — $ 229,484 Cash and cash equivalents Restricted cash 3,152 — — 3,152 Other non-current assets Total assets $ 232,636 $ — $ — $ 232,636 Liabilities: Contingent consideration — — 40,738 40,738 Contingent consideration Consideration for post-acquisition services — — 478 478 Other non-current liabilities Total liabilities $ — $ — $ 41,216 $ 41,216
Schedule of changes in fair value of contingent consideration	Amount of contingent consideration 2023 (in thousands) Balance at December 31, 2022 $ 35,316 Change in fair value (presented within research and development expenses) 15,441 Contingent consideration milestone payment (9,563) Currency translation effects (456) Balance at September 30, 2023 $ 40,738

Accrued expenses and other cu_2

Accrued expenses and other current liabilities (Tables)	9 Months Ended
Accrued expenses and other current liabilities (Tables)	Sep. 30, 2023
Accrued expenses and other current liabilities
Schedule of accrued expenses and other current liabilities	September 30, December 31, 2023 2022 (in thousands) Accruals for goods received from and services provided by vendors-not yet billed $ 11,688 $ 11,120 Personnel related accruals and liabilities 16,244 17,201 Liability owed to the Purchaser pursuant to the Royalty Financing Agreement 495 — Accrued contract fulfillment costs and costs to obtain a contract — 2,250 Total $ 28,427 $ 30,571

Royalty financing agreement (Ta

Royalty financing agreement (Tables)	9 Months Ended
Royalty financing agreement (Tables)	Sep. 30, 2023
Royalty Financing Agreement
Schedule of movement in the liability related to royalty financing agreement	Amount of liability (in thousands) Gross proceeds from royalty financing agreement on June 5, 2023 $ 375,000 Debt issuance costs paid (4,938) Royalty payments to Purchaser (796) Liability owed to the Purchaser (presented as "Accrued expense and other current liabilities") (495) Interest expense for the period June 5, 2023 to September 30, 2023 14,940 Liability related to the royalty financing agreement $ 383,711

Share-based compensation (Table

Share-based compensation (Tables)	9 Months Ended
Share-based compensation (Tables)	Sep. 30, 2023
Share-based compensation
Schedule of share-based compensation expense by classification included in consolidated statements of operations and comprehensive loss	Three months ended September 30, Nine months ended September 30, 2023 2022 2023 2022 (in thousands) (in thousands) Cost of manufacturing services revenue $ 315 $ 120 $ 477 $ 268 Research and development 5,638 3,730 14,675 12,090 Selling, general and administrative 5,144 3,758 12,900 9,932 Total $ 11,097 $ 7,608 $ 28,052 $ 22,290
Schedule of share-based compensation expense	Three months ended September 30, Nine months ended September 30, 2023 2022 2023 2022 (in thousands) (in thousands) Award type/ESPP Share options $ 3,497 $ 3,448 $ 10,256 $ 10,141 Restricted share units 5,293 3,957 15,311 11,354 Performance share units 2,299 197 2,461 777 Employee share purchase plan 8 6 24 18 Total $ 11,097 $ 7,608 $ 28,052 $ 22,290
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 $ 27,765 2.59 Restricted share units 35,763 2.03 Performance share units 556 1.20 Total $ 64,084 2.27
Schedule of weighted-average assumptions for fair value of option issued	Three months ended September 30, Nine months ended September 30, Assumptions 2023 2022 2023 2022 Expected volatility 70% 70% 70% 70% Expected terms 10 years 10 years 10 years 10 years Risk free interest rate 3.71% - 4.00% 4.16% 3.71% - 4.10% 2.12% - 4.16% Expected dividend yield 0% 0% 0% 0%
Summary of RSUs activity	RSUs Weighted average Number of grant-date fair ordinary shares value Non-vested at December 31, 2022 1,818,774 $ 20.46 Granted 1,614,925 $ 18.90 Vested (722,977) $ 22.57 Forfeited (224,402) $ 19.59 Non-vested at September 30, 2023 2,486,320 $ 18.91 Total weighted average grant date fair value of RSUs granted during the period (in $ millions) $ 30.5
Summary of PSUs activity	PSUs Weighted average Number of grant-date fair ordinary shares value Non-vested at December 31, 2022 400,690 $ 28.82 Vested (94,510) $ 30.65 Forfeited (42,745) $ 28.04 Non-vested at September 30, 2023 263,435 $ 28.29
2014 Plan.
Share-based compensation
Summary of option activity	Options Number of Weighted average ordinary shares exercise price Outstanding at December 31, 2022 4,237,917 $ 26.13 Granted 1,575,430 $ 18.69 Forfeited (257,880) $ 22.82 Expired (131,026) $ 43.95 Exercised (12,882) $ 9.58 Outstanding at September 30, 2023 5,411,559 $ 23.73 Thereof, fully vested, and exercisable on September 30, 2023 2,826,806 $ 26.43 Thereof, outstanding and expected to vest after September 30, 2023 2,584,753 $ 20.76 Total weighted average grant date fair value of options issued during the period (in $ millions) $ 17.1 Proceeds from option sales during the period (in $ millions) $ 0.1

Basic and diluted earnings pe_2

Basic and diluted earnings per share (Tables)	9 Months Ended
Basic and diluted earnings per share (Tables)	Sep. 30, 2023
Basic and diluted earnings per share
Schedule of potential dilutive common shares	Three months ended September 30, Nine months ended September 30, 2023 2022 2023 2022 Anti-dilutive ordinary share equivalents Stock options under 2014 Plans and previous plan 5,411,599 4,284,656 5,411,599 4,284,656 Non-vested RSUs and PSUs 2,749,755 2,328,151 2,749,755 2,328,151 ESPP 2,857 721 2,857 721 Total anti-dilutive ordinary share equivalents 8,164,211 6,613,528 8,164,211 6,613,528

CSL Behring collaboration - Nar

CSL Behring collaboration - Narrative (Details) - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2023	Sep. 30, 2022	Sep. 30, 2023	Sep. 30, 2022	Dec. 31, 2022
Collaboration arrangements
Revenue	$ 1,407	$ 1,449	$ 9,154	$ 3,738
CSL Behring collaboration.
Collaboration arrangements
Accounts receivable	1,600		1,600		$ 2,200
Contract asset					$ 100,000
License Revenue
Collaboration arrangements
Revenue	$ 497	$ 0	$ 1,290	$ 0

Investment securities (Details)

Investment securities (Details) - USD ($) $ in Thousands	9 Months Ended
Investment securities (Details) - USD ($) $ in Thousands	Sep. 30, 2023	Dec. 31, 2022
Current investments:
Amortized cost, as adjusted	$ 429,428	$ 124,831
Gross unrealized holding losses	(556)
Estimated fair value	428,872
Non-current investments:
Amortized cost, as adjusted		39,984
Investment securities
Amortized cost, as adjusted		164,815
Gross unrealized holding losses		(326)
Estimated fair value		164,489
Investment purchased	366,439
Obligations of governmental agencies
Current investments:
Amortized cost, as adjusted	429,428	124,831
Gross unrealized holding losses	(556)	(283)
Estimated fair value	$ 428,872	124,548
Non-current investments:
Amortized cost, as adjusted		39,984
Gross unrealized holding losses		(43)
Estimated fair value		$ 39,941

Inventories (Details)

Inventories (Details) - USD ($) $ in Thousands	Sep. 30, 2023	Dec. 31, 2022
Inventories
Raw materials	$ 7,426	$ 3,584
Work in progress	6,597	1,874
Finished goods	1,631	1,466
Inventories	$ 15,654	$ 6,924

Fair value measurement - Assets

Fair value measurement - Assets and liabilities measured on a recurring basis (Details) - USD ($) $ in Thousands	Sep. 30, 2023	Dec. 31, 2022	Sep. 30, 2022
Assets:
Restricted cash	$ 3,152		$ 3,106
Recurring Fair Value Measurements
Assets:
Cash and cash equivalents	229,484	$ 228,012
Restricted cash	3,152	3,161
Total assets	232,636	231,173
Liabilities:
Contingent consideration	40,738	35,316
Consideration for post-acquisition services	478	297
Total liabilities	41,216	35,613
Recurring Fair Value Measurements \| Fair value hierarchy Level 1
Assets:
Cash and cash equivalents	229,484	228,012
Restricted cash	3,152	3,161
Total assets	232,636	231,173
Recurring Fair Value Measurements \| Fair value hierarchy Level 3
Liabilities:
Contingent consideration	40,738	35,316
Consideration for post-acquisition services	478	297
Total liabilities	$ 41,216	$ 35,613

Fair value measurement - Contin

Fair value measurement - Contingent Consideration (Details) $ in Thousands, € in Millions		1 Months Ended		3 Months Ended		9 Months Ended			12 Months Ended
	Aug. 01, 2023	Aug. 31, 2023 USD ($)	Aug. 31, 2023 EUR (€)	Sep. 30, 2023 USD ($)	Sep. 30, 2022 USD ($)	Sep. 30, 2023 USD ($)	Sep. 30, 2023 EUR (€)	Sep. 30, 2022 USD ($)	Dec. 31, 2022 USD ($)	Sep. 30, 2023 EUR (€)
Fair Value, Liabilities Measured on Recurring Basis, Unobservable Input Reconciliation [Line Items]
Contingent consideration, net of current portion				$ 14,030		$ 14,030			$ 9,334
Contingent consideration				40,700		40,700
Current portion of contingent consideration				26,708		$ 26,708			25,982
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%
Change in fair value (presented within research and development expenses)						$ 72,000
uniQure France [S.A.S]
Fair Value, Liabilities Measured on Recurring Basis, Unobservable Input Reconciliation [Line Items]
Contingent consideration, net of current portion				40,738		40,738			$ 35,316
Milestone payment achieving due to increase In Probability Of Success In Clinical Development		$ 31,700	€ 30
Change in fair value (presented within research and development expenses)				14,200	$ 5,500	15,441		$ 7,500
Contingent consideration and milestone payment						10,600	€ 10
Contingent consideration milestone payment						9,563	€ 8.9
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				$ 189,100		$ 189,100				€ 178.8
Contingent consideration discount rate percentage						15.50%	15.50%		14.40%
Percentage of increase in Probability of Success	100%
uniQure France [S.A.S] \| Minimum
Fair Value, Liabilities Measured on Recurring Basis, Unobservable Input Reconciliation [Line Items]
Contingent consideration discount rate percentage						15%	15%		14%
Percentage of increase in Probability of Success		66%	66%

Fair value measurement - Change

Fair value measurement - Changes in fair value of contingent consideration (Details) $ in Thousands, € in Millions	3 Months Ended		9 Months Ended
	Sep. 30, 2023 USD ($)	Sep. 30, 2022 USD ($)	Sep. 30, 2023 USD ($)	Sep. 30, 2023 EUR (€)	Sep. 30, 2022 USD ($)
Fair Value, Liabilities Measured on Recurring Basis, Unobservable Input Reconciliation [Line Items]
Beginning Balance			$ 9,334
Ending Balance	$ 14,030		14,030
uniQure France [S.A.S]
Fair Value, Liabilities Measured on Recurring Basis, Unobservable Input Reconciliation [Line Items]
Beginning Balance			35,316
Change in fair value (presented within research and development expenses)	14,200	$ 5,500	15,441		$ 7,500
Contingent consideration milestone payment			9,563	€ 8.9
Currency translation effects			(456)
Ending Balance	$ 40,738		$ 40,738

Accrued expenses and other cu_3

Accrued expenses and other current liabilities (Details) - USD ($) $ in Thousands	Sep. 30, 2023	Dec. 31, 2022
Accrued expenses and other current liabilities
Accruals for goods received from and services provided by vendors-not yet billed	$ 11,688	$ 11,120
Personnel related accruals and liabilities	16,244	17,201
Liability owed to the Purchaser pursuant to the Royalty Financing Agreement	495
Accrued contract fulfillment costs and costs to obtain a contract		2,250
Total	$ 28,427	$ 30,571

Long-term debt (Details)

Long-term debt (Details) - USD ($) $ in Thousands		1 Months Ended	3 Months Ended		9 Months Ended
Long-term debt (Details) - USD ($) $ in Thousands	Apr. 01, 2024	Jun. 30, 2023	Sep. 30, 2023	Sep. 30, 2022	Sep. 30, 2023	Sep. 30, 2022	Jan. 05, 2027	Dec. 01, 2025	Dec. 31, 2022
Long-term Debt
Investment, Variable Interest Rate, Type [Extensible Enumeration]			us-gaap:PrimeRateMember		us-gaap:PrimeRateMember
Foreign currency gain / (loss)			$ 186	$ 14,362	$ (1,809)	$ 42,328
2023 Amended Facility
Long-term Debt
Interest rate (as a percent)			7.95%		7.95%
Variable interest rate basis					7.95%
Discount rate (as a percent)			3.25%		3.25%
Back-end fees paid		$ 2,500
Amortized cost net of discount and debt issuance costs			$ 102,500		$ 102,500				$ 103,800
Foreign currency gain / (loss)			(3,000)	(6,600)	(1,400)	(15,000)
Interest expense recorded			3,700	$ 3,000	10,900	$ 8,100
2023 Amended Facility \| Forecast
Long-term Debt
Back-end fees							$ 1,300
Restated Facility 2021
Long-term Debt
Outstanding debt			$ 100,000		$ 100,000
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					$ 871,600
Assets not being pledged to secure facilities by directly or indirectly					1,000
Venture debt loan facility \| 2023 Amended Facility \| uniQure France
Long-term Debt
Assets not being pledged to secure facilities by directly or indirectly					$ 85,000
Subsequent events. \| 2023 Amended Facility \| Forecast
Long-term Debt
Back-end fees								$ 4,900
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		4 Months Ended	9 Months Ended
Royalty financing agreement (Details) $ in Thousands	May 12, 2023 USD ($)	Sep. 30, 2023 USD ($)	Sep. 30, 2022 USD ($)	Sep. 30, 2023 USD ($)	Sep. 30, 2023 USD ($)	Sep. 30, 2022 USD ($)	Dec. 31, 2038	Jun. 30, 2032 USD ($)
Collaboration arrangements
Royalty financing agreement interest expense					$ 14,940
Net proceeds from the Royalty Financing Agreement					374,350
Payment of debt issuance costs					4,288
Interest Expense		$ 15,444	$ 3,069		25,846	$ 8,279
CLS
Collaboration arrangements
Maximum rights to all other royalties, as well as contractual milestones retained		1,300,000		$ 1,300,000	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		693,800	693,800
Royalty financing agreement interest expense				14,940	323,700
Net proceeds from the Royalty Financing Agreement					370,100
Payment of debt issuance costs				$ 4,938	4,900
Interest Expense		$ 11,800			$ 14,900
Royalty financing agreement \| Minimum
Collaboration arrangements
Effective interest rate (as a percent)		12%		12%	12%
Interest Expense		$ 11,500			$ 14,600
Royalty financing agreement \| Maximum
Collaboration arrangements
Effective interest rate (as a percent)		13.50%		13.50%	13.50%
Interest Expense		$ 13,000			$ 16,500
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 \| 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		$ 25,000	$ 25,000

Royalty financing agreement - M

Royalty financing agreement - Movement in the liability (Details) - USD ($) $ in Thousands		4 Months Ended	9 Months Ended
	Jun. 05, 2023	Sep. 30, 2023	Sep. 30, 2023
Collaboration arrangements
Debt issuance costs paid			$ 4,288
Liability owed to the Purchaser (presented as "Accrued expense and other current liabilities")		$ 495	495
Interest expense for the period			14,940
Liability related to the royalty financing agreement		383,711	383,711
Royalty financing agreement
Collaboration arrangements
Gross proceeds from royalty financing agreement	$ 375,000
Debt issuance costs paid		4,938	4,900
Royalty payments to Purchaser		(796)
Liability owed to the Purchaser (presented as "Accrued expense and other current liabilities")		495	495
Interest expense for the period		14,940	323,700
Liability related to the royalty financing agreement		$ 383,711	$ 383,711

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		9 Months Ended
	Jun. 30, 2018	Sep. 30, 2023	Sep. 30, 2022	Sep. 30, 2023	Sep. 30, 2022
2014 Plan
Share-based compensation
Authorized shares		12,601,471		12,601,471
Share-based compensation expense		$ 11,097	$ 7,608	$ 28,052	$ 22,290
Unrecognized compensation costs		64,084		$ 64,084
Weighted-average remaining period for recognition (in years)				2 years 3 months 7 days
2014 Plans and ESPP
Share-based compensation
Share-based compensation expense		11,097	7,608	$ 28,052	22,290
Research and development expenses \| 2014 Plans and ESPP
Share-based compensation
Share-based compensation expense		5,638	3,730	14,675	12,090
Selling, general and administrative expense \| 2014 Plans and ESPP
Share-based compensation
Share-based compensation expense		5,144	3,758	12,900	9,932
Cost of manufacturing services revenues \| 2014 Plans and ESPP
Share-based compensation
Share-based compensation expense		315	120	477	268
Share options \| 2014 Plan
Share-based compensation
Unrecognized compensation costs		27,765		$ 27,765
Weighted-average remaining period for recognition (in years)				2 years 7 months 2 days
Share options \| 2014 Plans and ESPP
Share-based compensation
Share-based compensation expense		3,497	3,448	$ 10,256	10,141
Restricted share units ("RSUs") \| 2014 Plan
Share-based compensation
Unrecognized compensation costs		35,763		$ 35,763
Weighted-average remaining period for recognition (in years)				2 years 10 days
Restricted share units ("RSUs") \| 2014 Plans and ESPP
Share-based compensation
Share-based compensation expense		5,293	3,957	$ 15,311	11,354
Performance Share Awards \| 2014 Plan
Share-based compensation
Unrecognized compensation costs		556		$ 556
Weighted-average remaining period for recognition (in years)				1 year 2 months 12 days
Performance Share Awards \| 2014 Plans and ESPP
Share-based compensation
Share-based compensation expense		$ 2,299	$ 197	$ 2,461	$ 777
Employee share purchase plan \| Employee share purchase plan (ESPP)
Share-based compensation
Ordinary shares available for issue	150,000	103,790	117,997	103,790	117,997
Discounted rate for purchase of shares	85%
Employee share purchase plan \| 2014 Plans and ESPP
Share-based compensation
Share-based compensation expense		$ 8	$ 6	$ 24	$ 18

Share-based compensation - Opti

Share-based compensation - Option activity and weighted-average assumptions (Details) - USD ($) $ / shares in Units, $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2023	Sep. 30, 2022	Sep. 30, 2023	Sep. 30, 2022
Weighted average exercise price
Total weighted average grant date fair value of options issued during the period (in $ millions)			$ 17,100
Proceeds from Issuance of Shares under Incentive and Share-based Compensation Plans, Including Stock Options			262	$ 844
Proceeds from option sales during the period (in $ millions)			$ 100
2014 Plan
Weighted average exercise price
Vesting period (in years)			4 years
2014 Plan \| Share options
Options
Outstanding at beginning of the period (in shares)			4,237,917
Granted (in shares)			1,575,430
Forfeited (in shares)			(257,880)
Expired (in shares)			(131,026)
Exercised (in shares)			(12,882)
Outstanding at end of the period (in shares)	5,411,559		5,411,559
Thereof, fully vested and exercisable at end of period (in shares)	2,826,806		2,826,806
Thereof, outstanding and expected to vest after at end of period (in shares)	2,584,753		2,584,753
Weighted average exercise price
Outstanding at beginning of the period (in dollars per share)			$ 26.13
Granted (in dollars per share)			18.69
Forfeited (in dollars per share)			22.82
Expired (in dollars per share)			43.95
Exercised (in dollars per share)			9.58
Outstanding at end of period (in dollars per share)	$ 23.73		23.73
Thereof, fully vested and exercisable at end of period (in dollars per share)	$ 26.43		26.43
Outstanding and expected to vest after at end of the period (in dollars per share)			$ 20.76
Weighted-average assumptions used to estimate fair value of share options granted during year
Expected volatility (as a percent)	70%	70%	70%	70%
Expected terms (in years)	10 years	10 years	10 years	10 years
Risk free interest rate, minimum (as a percent)	3.71%	4.16%	3.71%	2.12%
Risk free interest rate, maximum (as a percent)	4%		4.10%	4.16%
Expected dividend (as a percent)	0%	0%	0%	0%
2014 Plan \| Non-executive directors
Weighted average exercise price
Vesting period (in years)			1 year
2014 Plan \| 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) - 2014 Plan $ / shares in Units, $ in Millions	9 Months Ended
	Sep. 30, 2023 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	1,818,774
Granted (in shares) \| shares	1,614,925
Vested (in shares) \| shares	(722,977)
Forfeited (in shares) \| shares	(224,402)
Non-vested at end of period (in shares) \| shares	2,486,320
Weighted average grant-date fair value
Non-vested at beginning of period (in dollars per share) \| $ / shares	$ 20.46
Granted (in dollars per share) \| $ / shares	18.90
Vested (in dollars per share) \| $ / shares	22.57
Forfeited (in dollars per share) \| $ / shares	19.59
Non-vested at end of period (in dollars per share) \| $ / shares	$ 18.91
Other disclosure
Total weighted average grant date fair value of RSUs granted during the period (in millions) \| $	$ 30.5
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)	1 Months Ended	9 Months Ended	12 Months Ended
Share-based compensation - PSU activity (Details)	Sep. 30, 2021	Sep. 30, 2023 Milestone $ / shares shares	Dec. 31, 2022 $ / shares shares
Other disclosure
Service period	1 year		3 years
Performance Share Awards
Other disclosure
Number of Milestones Achieved \| Milestone		2
Performance Share Awards \| 2014 Plan
Number of shares
Non-vested at beginning of period (in shares) \| shares		400,690
Vested (in shares) \| shares		(94,510)
Forfeited (in shares) \| shares		(42,745)
Non-vested at end of period (in shares) \| shares		263,435	400,690
Weighted average grant-date fair value
Non-vested at beginning of period (in dollars per share) \| $ / shares		$ 28.82
Vested (in dollars per share) \| $ / shares		30.65
Forfeited (in dollars per share) \| $ / shares		28.04
Non-vested at end of period (in dollars per share) \| $ / shares		$ 28.29	$ 28.82

Share-based compensation - Empl

Share-based compensation - Employee Share Purchase Plan (Details) - Employee share purchase plan (ESPP) - Employee Stock - shares	9 Months Ended
	Sep. 30, 2023	Sep. 30, 2022	Jun. 30, 2018
Share-based compensation
Number of shares issued	12,270	9,305
Ordinary shares available for issue	103,790	117,997	150,000

Income taxes - Effective Tax Ra

Income taxes - Effective Tax Rate (Details) - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2023	Sep. 30, 2022	Sep. 30, 2023	Sep. 30, 2022
Effective income tax rate	(0.10%)	(0.70%)	(0.50%)	(0.90%)
Enacted tax rate			25.80%
Deferred tax loss (benefit)			$ (1,113)	$ (1,263)
United States and France
Deferred tax loss (benefit)	$ 100	$ 300	$ 1,100	$ 1,300

Basic and diluted earnings pe_3

Basic and diluted earnings per share (Details) - shares	3 Months Ended		9 Months Ended
Basic and diluted earnings per share (Details) - shares	Sep. 30, 2023	Sep. 30, 2022	Sep. 30, 2023	Sep. 30, 2022
Basic and diluted earnings per share
Total potential dilutive ordinary shares	8,164,211	6,613,528	8,164,211	6,613,528
ESPP
Basic and diluted earnings per share
Total potential dilutive ordinary shares	2,857	721	2,857	721
Employee Stock Option [Member]
Basic and diluted earnings per share
Total potential dilutive ordinary shares	5,411,599	4,284,656	5,411,599	4,284,656
Non-vested RSUs and PSUs
Basic and diluted earnings per share
Total potential dilutive ordinary shares	2,749,755	2,328,151	2,749,755	2,328,151

Subsequent events (Details)

Subsequent events (Details) - Subsequent events. - Employee severance pursuant to strategic reorganization plan - USD ($) $ in Millions	Oct. 05, 2023	Dec. 31, 2023
Subsequent Event [Line Items]
Percent of workforce reduction	20%
Estimated charges related to cash expenditures for employee severance costs		$ 2.3

Insider Trading Arrangements

Insider Trading Arrangements	3 Months Ended
Insider Trading Arrangements	Sep. 30, 2023
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