uniQure (QURE) 10-Q2 Nov 222022 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, 2022	Oct. 28, 2022
Document and Entity Information
Document Type	10-Q
Document Quarterly Report	true
Document Period End Date	Sep. 30, 2022
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		46,815,109
Entity Central Index Key	0001590560
Current Fiscal Year End Date	--12-31
Document Fiscal Year Focus	2022
Document Fiscal Period Focus	Q3
Amendment Flag	false

CONSOLIDATED BALANCE SHEETS

CONSOLIDATED BALANCE SHEETS - USD ($) $ in Thousands	Sep. 30, 2022	Dec. 31, 2021
Current assets
Cash and cash equivalents	$ 440,313	$ 556,256
Accounts receivable and contract asset	3,603	58,768
Inventories	4,075
Prepaid expenses	13,692	10,540
Other current assets and receivables	2,894	2,675
Total current assets	464,577	628,239
Non-current assets
Property, plant and equipment, net of accumulated depreciation of $40.5 million as of September 30, 2022 and $36.9 million as of December 31, 2021	47,886	43,505
Operating lease right-of-use assets	27,804	25,573
Intangible assets, net, including an in-process research and development asset of $52.5 million as of September 30, 2022 and $60.8 million as of December 31, 2021	53,837	62,686
Goodwill	23,418	27,633
Deferred tax assets, net	14,627	15,647
Other non-current assets	6,085	5,897
Total non-current assets	173,657	180,941
Total assets	638,234	809,180
Current liabilities
Accounts payable	7,566	2,502
Accrued expenses and other current liabilities	26,437	28,487
Current portion of contingent consideration	23,537
Current portion of operating lease liabilities	6,434	5,774
Total current liabilities	63,974	36,763
Non-current liabilities
Long-term debt	102,394	100,963
Operating lease liabilities, net of current portion	29,893	28,987
Contingent consideration, net of current portion	9,158	29,542
Deferred tax liability, net	8,592	12,913
Other non-current liabilities	3,053	4,236
Total non-current liabilities	153,090	176,641
Total liabilities	217,064	213,404
Commitments and contingencies
Shareholders' equity
Ordinary shares, €0.05 par value: 80,000,000 shares authorized as of September 30, 2022 and December 31, 2021 and 46,815,109 and 46,298,635 ordinary shares issued and outstanding as of September 30, 2022 and December 31, 2021, respectively	2,830	2,802
Additional paid-in-capital	1,100,078	1,076,972
Accumulated other comprehensive loss	(93,000)	(28,856)
Accumulated deficit	(588,738)	(455,142)
Total shareholders' equity	421,170	595,776
Total liabilities and shareholders' equity	$ 638,234	$ 809,180

CONSOLIDATED BALANCE SHEETS (Pa

CONSOLIDATED BALANCE SHEETS (Parenthetical) $ in Thousands	Sep. 30, 2022 USD ($) shares	Sep. 30, 2022 € / shares	Dec. 31, 2021 USD ($) shares	Dec. 31, 2021 € / shares
Accumulated depreciation \| $	$ 40,500		$ 36,900
Intangible assets, net \| $	$ 53,837		$ 62,686
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	46,815,109		46,298,635
Ordinary shares, outstanding \| shares	46,815,109		46,298,635
In-process research & development
Intangible assets, net \| $	$ 52,500		$ 60,800

CONSOLIDATED STATEMENTS OF OPER

CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE (LOSS) INCOME - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2022	Sep. 30, 2021	Sep. 30, 2022	Sep. 30, 2021
Total revenues	$ 1,449	$ 1,989	$ 3,738	$ 466,311
Operating expenses:
Cost of contract revenues				(23,178)
Cost of contract manufacturing	(861)		(1,693)
Research and development expenses	(48,068)	(36,432)	(139,263)	(101,209)
Selling, general and administrative expenses	(13,324)	(12,023)	(36,802)	(42,323)
Total operating expenses	(62,253)	(48,455)	(177,758)	(166,710)
Other income	1,485	1,680	4,981	9,622
Other expense	(199)	(214)	(621)	(673)
(Loss) / income from operations	(59,518)	(45,000)	(169,660)	308,550
Interest income	39	46	117	123
Interest expense	(3,069)	(1,924)	(8,279)	(5,377)
Foreign currency gains, net	14,362	10,436	42,328	21,645
Other non-operating (losses) / gains, net			635
(Loss) / income before income tax benefit / (expense)	(48,186)	(36,442)	(134,859)	324,941
Income tax benefit / (expense)	329	(89)	1,263	(3,560)
Net (loss) / income	(47,857)	(36,531)	(133,596)	321,381
Other comprehensive loss:
Foreign currency translation adjustments	(25,370)	(13,288)	(64,144)	(27,790)
Total comprehensive (loss) / gain	$ (73,227)	$ (49,819)	$ (197,740)	$ 293,591
Earnings per ordinary share - basic
Basic net (loss) / income per ordinary share	$ (1.02)	$ (0.79)	$ (2.86)	$ 7
Earnings per ordinary share - diluted
Diluted net (loss) / income per ordinary share	$ (1.02)	$ (0.79)	$ (2.86)	$ 6.87
Weighted average shares - basic	46,772,430	46,152,404	46,680,667	45,888,769
Weighted average shares - diluted	46,772,430	46,152,404	46,680,667	46,780,963
License revenues
Total revenues				$ 462,400
Collaboration revenues
Total revenues	$ 1,449	$ 1,989	$ 3,738	$ 3,911

CONSOLIDATED STATEMENTS OF SHAR

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, 2020	$ 2,711	$ 1,016,018	$ 9,907	$ (784,731)	$ 243,905
Beginning balance (in shares) at Dec. 31, 2020	44,777,799
Increase (decrease) in shareholders' equity
Income / Loss for the period				321,381	321,381
Other comprehensive loss			(27,790)		(27,790)
Issuance of ordinary shares	$ 55	29,510			29,565
Issuance of ordinary shares (in shares)	921,730
Income tax benefit of past share issuance cost		2,977			2,977
Exercise of share options	$ 10	1,814			1,824
Exercise of share options (in shares)	156,787
Restricted and performance share units distributed during the period	$ 21	(21)
Restricted and performance share units distributed during the period (in shares)	346,736
Share-based compensation expense		18,747			18,747
Issuance of ordinary shares relating to employee stock purchase plan		146			146
Issuance of ordinary shares relating to employee stock purchase plan (in shares)	4,724
Ending balance at Sep. 30, 2021	$ 2,797	1,069,191	(17,883)	(463,350)	590,755
Ending balance (in shares) at Sep. 30, 2021	46,207,776
Beginning balance at Jun. 30, 2021	$ 2,788	1,062,234	(4,595)	(426,819)	633,608
Beginning balance (in shares) at Jun. 30, 2021	46,050,250
Increase (decrease) in shareholders' equity
Income / Loss for the period				(36,531)	(36,531)
Other comprehensive loss			(13,288)		(13,288)
Exercise of share options	$ 5	909			914
Exercise of share options (in shares)	84,811
Restricted and performance share units distributed during the period	$ 4	(4)
Restricted and performance share units distributed during the period (in shares)	69,165
Share-based compensation expense		5,955			5,955
Issuance of ordinary shares relating to employee stock purchase plan		97			97
Issuance of ordinary shares relating to employee stock purchase plan (in shares)	3,550
Ending balance at Sep. 30, 2021	$ 2,797	1,069,191	(17,883)	(463,350)	590,755
Ending balance (in shares) at Sep. 30, 2021	46,207,776
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				46,298,635
Increase (decrease) in shareholders' equity
Income / 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				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
Income / 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				46,815,109

CONSOLIDATED STATEMENTS OF CASH

CONSOLIDATED STATEMENTS OF CASH FLOWS - USD ($) $ in Thousands	9 Months Ended
	Sep. 30, 2022	Sep. 30, 2021
Cash flows from operating activities
Net (loss) / income	$ (133,596)	$ 321,381
Adjustments to reconcile net (loss) / income to net cash (used in) / generated from operating activities:
Depreciation and amortization expense	6,206	5,447
Share-based compensation expense	22,290	18,747
Deferred tax (income) / expense	(1,263)	3,560
Changes in fair value of contingent consideration and derivative financial instrument, net	6,875	416
Unrealized foreign exchange gains, net	(36,390)	(23,242)
Other non-current assets, net	(581)	(2,862)
Changes in operating assets and liabilities:
Accounts receivable and contract asset, prepaid expenses, and other current assets and receivables	41,992	(1,993)
Inventories	(4,075)
Accounts payable	4,529	2,003
Accrued expenses, other liabilities, and operating leases	3,633	11,592
Net cash (used in) / generated from operating activities	(90,380)	335,049
Cash flows from investing activities
Purchases of property, plant, and equipment	(12,622)	(13,885)
Acquisition of Corlieve, net of cash acquired	(1,900)	(49,949)
Net cash used in investing activities	(14,522)	(63,834)
Cash flows from financing activities
Proceeds from issuance of ordinary shares related to employee stock option and purchase plans	844	1,971
Proceeds from loan increment, net of debt issuance costs		34,603
Proceeds from issuance of ordinary shares		30,899
Share issuance costs from issuance of ordinary shares		(1,334)
Repayment of debt acquired through acquisition of Corlieve		(1,175)
Net cash generated from financing activities	844	64,964
Currency effect on cash, cash equivalents and restricted cash	(11,876)	(2,589)
Net (decrease) / increase in cash, cash equivalents and restricted cash	(115,934)	333,590
Cash, cash equivalents and restricted cash at beginning of period	559,353	247,680
Cash, cash equivalents and restricted cash at the end of period	443,419	581,270
Cash and cash equivalents	440,313	578,464
Restricted cash related to leasehold and other deposits	3,106	2,806
Total cash, cash equivalents and restricted cash	443,419	581,270
Supplemental cash flow disclosures:
Cash paid for interest	(6,410)	(4,503)
Non-cash (decrease) / increase in accounts payables and accrued expenses and other current liabilities related to purchases of property, plant, and equipment	$ 1,766	$ 415

General business information

General business information	9 Months Ended
General business information	Sep. 30, 2022
General business information
General business information	1 uniQure (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 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, 2022
Summary of significant accounting policies
Summary of significant accounting policies	2 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 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 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, 2022, are not necessarily indicative of the results to be expected for the full year ending December 31, 2022, 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, 2021, and the notes thereto, which are included in the Company’s Annual Report Inventories The Company started producing commercial materials in April 2022 to supply CSL Behring LLC (“CSL Behring”) with etranacogene dezaparvovec (the “Product”) in accordance with the June 2020 Development and Commercial Supply Agreement between the Company and CSL Behring. From this date onwards, the Company presents the costs associated with the aforementioned activities as cost of contract manufacturing. The Company capitalizes inventory to be sold to CSL Behring to the extent it expects to generate probable future benefits from such sales. Refer to Note 3, “CSL Behring collaboration” Per ASC 330, Inventory, inventory is stated at the lower of cost or estimated net realizable value, on a first-in, first-out basis. The Company capitalizes raw materials to the extent these can be used in the manufacturing of the Product. The Company uses standard costs, approximating average costs to determine its cost basis for work in progress and finished goods. The Company’s assessment of recoverability value requires the use of estimates regarding the net realizable value of its inventory balances, including an assessment of excess or obsolete inventory. As applicable, write-downs resulting from adjustments to net realizable value will be recorded to cost of contract manufacturing. 2.5 Recent accounting pronouncements There have been no new accounting pronouncements or changes to accounting pronouncements during the nine months ended September 30, 2022, as compared to the recent accounting pronouncements described in Note 2.3.23 of the Company’s Annual Report

CSL Behring collaboration

CSL Behring collaboration	9 Months Ended
CSL Behring collaboration	Sep. 30, 2022
CSL Behring collaboration
CSL Behring collaboration	3 CSL Behring collaboration On June 24, 2020 (the “Signing Date”), uniQure biopharma B.V., a wholly owned subsidiary of uniQure N.V., entered into a commercialization and license agreement (the “CSL Behring Agreement”) with CSL Behring, pursuant to which CSL Behring received exclusive global rights to etranacogene dezaparvovec, the Company’s investigational gene therapy for patients with hemophilia B. The transaction became fully effective on May 6, 2021, one day after the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976 (the “HSR Act”) expired on May 5, 2021 (“Closing”). The Company, as of the effective date, identified two material performance obligations related to the CSL Behring Agreement: (i) Sale of the exclusive global rights to the Product (“License Sale”); and (ii) Generate information to support the regulatory approval of the current and next generation manufacturing process of the Product and to provide any such information generated to CSL Behring (“Manufacturing Development”). License Sale The Company continued to develop the Product between the Signing Date and Closing and performed certain reimbursable activities to fulfill the transfer of the global rights (“Additional Covenants” that are included as part of the “License Sale”). The Additional Covenants are not considered distinct from the performance obligation to sell the license to CSL Behring as CSL Behring could not benefit from the Additional Covenants on their own, or have these activities be performed with readily available resources. The Company determined that the fixed upfront payment of $450.0 million and the $12.4 million that the Company received in relation to the Additional Covenants should be allocated to the License Sale. The Company determined that the License Sale was completed on May 6, 2021, when it transferred the license and CSL Behring assumed full responsibility for the development and commercialization of the Product. At Closing, the Company evaluated the amounts of potential payments and the likelihood that the payments will be received. The Company utilized the most likely amount method to estimate the variable consideration to be included in the transaction price. Since the Company cannot control the achievement of regulatory and first commercial sales milestones, the Company concluded that the potential payments are constrained as of Closing. The Company determined that it would recognize revenue related to these payments only to the extent that it becomes probable that no significant reversal of recognized cumulative revenue will occur thereafter. Similarly, the Company records expenses related to its existing license and other agreements as well as its financial advisor for a mid-single digit percentage of any such revenue recognized associated to meeting a milestone. The Company includes payments related to sales milestones in the transaction price when their achievement becomes probable, and it will include royalties on the sale of the Product once these have been earned. The Company recognized nil and $462.4 million of license revenue in the three and nine months ended September 30, 2021 related to the License Sale. During the three and nine months ended September 30, 2022, the Company did no t recognize any license revenue. Collaboration Services The Company recognized $0.7 million and $2.1 million of collaboration revenue in the three and nine months ended September 30, 2022, compared to $0.5 and $0.9 million in the three and nine months ended September 30, 2021. The Company generates such collaboration revenue from services rendered in relation to completing the HOPE-B clinical trial on behalf of CSL Behring as well as additional development services that CSL Behring requested. These collaboration services are reimbursed at the pre-agreed full-time-employee rate (“FTE-rate”). Contract Manufacturing The Company commenced capitalizing inventory in April 2022 for the Product to be sold to CSL Behring. On September 6, 2022, CSL Behring notified the Company of its intent to transfer manufacturing technology related to the Product to a third-party contract manufacturer designated by CSL Behring. CSL Behring also requested that the Company continue to serve as a manufacturer of the Product after the Company completes the technology transfer to a third party. The Company and CSL Behring are in the process of negotiating the terms of the transfer of manufacturing responsibility pursuant to the CSL Behring Agreement. Accounts Receivable and Contract Asset As of December 31, 2021, the Company recorded accounts receivable of $2.9 million from CSL Behring related to collaboration services as well as a contract asset of $55.0 million associated with milestone payments due upon CSL Behring’s global regulatory submissions for etranacogene dezaparvovec, which were deemed to be probable. In March and April 2022, CSL Behring submitted marketing applications in the United States and European Union, and as of March and April 2022, the Company had collected the $55.0 million owed. As of September 30, 2022, the Company had accounts receivable of $3.0 million from CSL Behring.

Inventories

Inventories	9 Months Ended
Inventories	Sep. 30, 2022
Inventories
Inventories	4 Inventories The Company commenced capitalizing inventory in April 2022 when the Company started producing commercial materials to supply the Product to CSL Behring. The following table summarizes the inventory balances for the nine months ended September 30, 2022: September 30, December 31, 2022 2021 (in thousands) Raw materials $ 1,447 $ — Work in progress 2,178 — Finished goods 450 — Inventories $ 4,075 $ —

Fair value measurement

Fair value measurement	9 Months Ended
Fair value measurement	Sep. 30, 2022
Fair value measurement
Fair value measurement	5 Fair value measurement The Company measures certain financial assets and liabilities at fair value, either upon initial recognition or for subsequent accounting or reporting. U.S. GAAP requires disclosure of methodologies used in determining the reported fair values and establishes a hierarchy of inputs used when available. The three levels of the fair value hierarchy are described below: Level 1 – Valuations based on unadjusted quoted prices in active markets for identical assets or liabilities that the Company can access at the measurement date. Level 2 – Valuations based on quoted prices for similar assets or liabilities in markets that are not active or models for which the inputs are observable, either directly or indirectly. Level 3 – Valuations that require inputs that reflect the Company’s own assumptions that are both significant to the fair value measurement and are unobservable. To the extent that valuation is based on models or inputs that are less observable or unobservable in the market, the determination of fair value requires more judgment. Accordingly, the degree of judgment exercised by the Company in determining fair value is greatest for instruments categorized as Level 3. A financial instrument’s level within the fair value hierarchy is based on the lowest level of any input that is significant to the fair value measurement. The carrying amount of cash and cash equivalents, accounts receivable, prepaid expenses, 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, 2022, and December 31, 2021: 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, 2021 Assets: Cash and cash equivalents $ 556,256 $ — $ — $ 556,256 Cash and cash equivalents Restricted cash 3,097 — — 3,097 Other non-current assets Total assets $ 559,353 $ — $ — $ 559,353 Liabilities: Contingent consideration — — 29,542 29,542 Contingent consideration Derivative financial instrument — — 2,805 2,805 Other non-current liabilities Consideration for post-acquisition services — — 846 846 Other non-current liabilities Total liabilities $ — $ — $ 33,193 $ 33,193 At September 30, 2022 Assets: Cash and cash equivalents $ 440,313 $ — $ — $ 440,313 Cash and cash equivalents Restricted cash 3,106 — — 3,106 Other non-current assets Total assets $ 443,419 $ — $ — $ 443,419 Liabilities: Contingent consideration — — 32,695 32,695 Current portion of contingent consideration; contingent consideration, net of current portion Derivative financial instrument — — 2,171 2,171 Other non-current liabilities Consideration for post-acquisition services — — 312 312 Other non-current liabilities Total liabilities $ — $ — $ 35,178 $ 35,178 Contingent consideration The Company is required to pay up to EUR 178.8 million ($175.2 million) at September 30, 2022 to the former shareholders of Corlieve upon the achievement of contractually defined milestones in connection with the Company’s acquisition of Corlieve. The fair market value of the contingent consideration was determined using unobservable initial inputs with respect to (i) the probability of achieving the relevant milestones, or POS, (ii) the estimated timing of achieving such milestones, and (iii) the interest rate used to discount the payments. The Company determined the fair market value of the contingent consideration by calculating the probability-adjusted payments based on each milestone’s probability of achievement. The probability-adjusted payments were then discounted to present value using a discount rate representing the Company’s credit risk. The discount rate was determined using the effective interest rate of the Company’s existing debt facility adjusted for difference in maturity dates based on market data on effective yields for US bonds with a CCC credit rating. The following table presents the changes in fair value of contingent consideration between December 31, 2021 and September 30, 2022: Amount of contingent consideration 2022 (in thousands) Balance at December 31, 2021 $ 29,542 Change in fair value (presented within research and development expenses) 7,510 Currency translation effects (4,357) Balance at September 30, 2022 $ 32,695 As of September 30, 2022, the Company classified $23.5 million of the total contingent consideration of $32.7 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. Derivative financial instrument The Company issued a derivative financial instrument related to its collaboration with BMS. On December 1, 2020, the Company and BMS agreed that upon the consummation of a change of control transaction of uniQure that occurs prior to December 1, 2026 or BMS’ delivery of a target cessation notice for all four collaboration targets, the Company (or its third party acquirer) shall pay to BMS a one-time, non-refundable, non-creditable cash payment of $70.0 million, provided that (x) if $70.0 million is greater than five percent (5.0%) of the net proceeds (as contractually defined) from such change of control transaction, the payment shall be an amount equal to five percent of such net proceeds, and (y) if $70.0 million is less than one percent of such net proceeds, the change of control payment shall be an amount equal to one percent of such net proceeds (“CoC-payment”). The Company has not consummated any change of control transaction as of September 30, 2022 that would obligate it to make a CoC-payment. The Company determined that the CoC-payment should be recorded as a derivative financial liability as of December 1, 2020 and that subsequent changes in the fair market value of this derivative financial liability should be recorded in profit and loss. The fair market value of the derivative financial liability is materially impacted by probability that market participants assign to the likelihood of the occurrence of a change of control transaction that would give rise to a CoC-payment. This probability represents an unobservable input. The Company determines the fair market value of the derivative financial liability by using a present value model based on expected cash flow. The expected cash flows are materially impacted by the probability that market participants assign to the likelihood of the occurrence of a change of control transaction within the biotechnology industry. The Company estimated this unobservable input using the best information available as of September 30, 2022 and December 31, 2021. The Company obtained reasonably available market information that it believed market participants would use in determining the likelihood of the occurrence of a change-of control transaction within the biotechnology industry. Selecting and evaluating market information involves considerable judgement and uncertainty. Based on all such information and its judgment, the Company estimated that the fair market value of the derivative financial liability (presented within “Other non-current liabilities”) as of September 30, 2022 was $2.2 million and as of December 31, 2021 was $2.8 million. The change in the fair market value of the derivative financial liability was nil for the three months ended September 30, 2022 and was $0.6 million for the nine months ended September 30, 2022 related to a decrease in the fair market value (nil in the same periods in 2021). The decrease in fair market value for the nine months ended September 30, 2022 was recorded as a gain within Other non-operating (losses) / gains

Accrued expenses and other curr

Accrued expenses and other current liabilities	9 Months Ended
Accrued expenses and other current liabilities	Sep. 30, 2022
Accrued expenses and other current liabilities
Accrued expenses and other current liabilities	6 Accrued expenses and other current liabilities Accrued expenses and other current liabilities include the following items: September 30, December 31, 2022 2021 (in thousands) Accruals for goods received from and services provided by vendors-not yet billed $ 13,497 $ 13,012 Personnel related accruals and liabilities 12,940 12,603 Accrued contract fulfillment costs and costs to obtain a contract — 2,872 Total $ 26,437 $ 28,487

Long-term debt

Long-term debt	9 Months Ended
Long-term debt	Sep. 30, 2022
Long-term debt
Long-term debt	7 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 (“2018 Amended Facility”), January 2021 (“2021 Amended Facility”) and in December 2021 (“2021 Restated Facility”). On January 29, 2021, the Company drew down $35.0 million as part of the amendment of the facility at that time. Pursuant to the 2021 Restated Facility, Tranche A and Tranche B of the 2021 Amended Facility with a total outstanding balance of $70.0 million were consolidated into one tranche with a total commitment of $100.0 million. The Company drew down an additional $30.0 million at the time of the December 2021 amendment, resulting in total principal outstanding as of September 30, 2022 of $100.0 million. The 2021 Restated Facility extended the loan’s maturity date from June 1, 2023 until December 1, 2025. The interest-only period was extended from January 1, 2023 to December 1, 2024, or December 1, 2025 if, prior to June 30, 2024, either (a) the Biologics License Application (“BLA”) for AMT-061 is approved by the U.S. Food and Drug Administration (“FDA”) or (b) AMT-130 is advanced into a pivotal trial. The interest rate is adjustable and is the greater of (i) 7.95% and (ii) 7.95% plus the prime rate less 3.25% per annum. Under the 2021 Restated Facility, the Company owes a back-end fee of 4.85% of the outstanding debt. The Company is required to repay the facility in equal monthly installments of principal and interest between the end of the interest-only period and the maturity date. The Company continues to owe a $2.5 million back-end fee related to the 2021 Amended Facility which is due on June 1, 2023. The amortized cost (including interest due presented as part of accrued expenses and other current liabilities) of the 2021 Restated Facility was $103.3 million as of September 30, 2022, compared to $101.6 million as of December 31, 2021, 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, 2022 was $6.6 million and $15.0 million, respectively compared to a foreign currency loss of $1.8 million and $4.0 million during the same periods in 2021 for the 2018 Amended Facility and the 2021 Amended Facility. Interest expense associated with the 2021 Restated Facility during the three and nine months ended September 30, 2022 was $3.0 million and $8.1 million, respectively compared to $1.9 million and $5.2 million, respectively, during the same periods in 2021 for the 2018 Amended Facility and the 2021 Amended Facility. As a covenant in the 2021 Restated Facility the Company has periodic reporting requirements and is required to keep a minimum cash balance deposited in bank accounts in the United States 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. The Company, beginning on April 1, 2023, is also required to keep a minimum of unrestricted cash of at least 50% of the loan amount outstanding. If, prior to June 30, 2024, either (a) the BLA for AMT-061 is approved by the FDA or (b) AMT-130 is advanced into a pivotal trial, the minimum cash covenant will be lowered to at least 30% of the loan amount outstanding and its effectiveness will be deferred to April 1, 2024. In combination with other covenants, the 2021 Restated 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 $638.2 million, less $75.3 million of cash and cash equivalents and other current assets held by uniQure N.V. and $80.2 million of other current assets and investment held by Corlieve Therapeutics SAS. The 2021 Restated Facility contains provisions that include the occurrence of a material adverse effect, as defined therein, which would entitle Hercules to declare all principal, interest and other amounts owed by the Company immediately due and payable. As of September 30, 2022, the Company was in material compliance with all covenants and provisions.

Share-based compensation

Share-based compensation	9 Months Ended
Share-based compensation	Sep. 30, 2022
Share-based compensation
Share-based compensation	8 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”). At the annual general meeting of shareholders in June 2021, the Company’s shareholders approved an increase in the number of ordinary shares authorized for issuance under the 2014 Plan from 8,601,471 to 12,601,471 . In June 2018, the Company’s shareholders adopted and approved an employee share purchase plan (“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. 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, 2022 2021 2022 2021 (in thousands) (in thousands) Research and development $ 3,730 $ 3,249 $ 12,090 $ 9,222 Selling, general and administrative 3,758 2,707 9,932 9,525 Cost of contract manufacturing 120 — 268 — Total $ 7,608 $ 5,956 $ 22,290 $ 18,747 Share-based compensation expense recognized by award type of the 2014 Plans as well as the ESPP was as follows: Three months ended September 30, Nine months ended September 30, 2022 2021 2022 2021 (in thousands) (in thousands) Award type/ESPP Share options $ 3,448 $ 2,965 $ 10,141 $ 9,155 Restricted share units 3,957 2,582 11,354 8,364 Performance share units 197 403 777 1,206 ESPP 6 6 18 22 Total $ 7,608 $ 5,956 $ 22,290 $ 18,747 As of September 30, 2022, 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 $ 27,720 2.64 Restricted share units 28,629 2.04 Total $ 56,349 2.34 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% 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. Options Number of Weighted average ordinary shares exercise price Outstanding at December 31, 2021 3,308,325 $ 31.02 Granted 1,399,466 $ 15.75 Forfeited (183,870) $ 38.74 Expired (133,381) $ 35.79 Exercised (115,884) $ 6.03 Outstanding at September 30, 2022 4,274,656 $ 26.22 Thereof, fully vested and exercisable on September 30, 2022 2,068,810 $ 28.33 Thereof, outstanding and expected to vest after September 30, 2022 2,205,846 $ 24.24 Total weighted average grant date fair value of options issued during the period (in $ millions) $ 12.5 Proceeds from option sales during the period (in $ millions) $ 0.7 The fair value of each option issued is estimated at the respective grant date using the Hull & White option pricing model with the following weighted-average assumptions: Three months ended September 30, Nine months ended September 30, Assumptions 2022 2021 2022 2021 Expected volatility 70% 75% 70% 75% Expected terms 10 years 10 years 10 years 10 years Risk free interest rate 4.16% 1.47% 2.12% - 4.16% 1.21 - 1.85% Expected dividend yield 0% 0% 0% 0% Restricted share units (“RSUs”) The following table summarizes the RSUs activity for the nine months ended September 30, 2022: RSU Weighted average Number of grant-date fair ordinary shares value Non-vested at December 31, 2021 710,617 $ 38.89 Granted 1,562,133 $ 15.91 Vested (283,349) $ 39.47 Forfeited (176,110) $ 23.76 Non-vested at September 30, 2022 1,813,291 $ 20.47 Total weighted average grant date fair value of RSUs granted during the period (in $ millions) $ 24.8 RSUs vest over one Performance share units (“PSUs”) The following table summarizes the PSUs activity for the nine months ended September 30, 2022: PSU Weighted average Number of grant-date fair ordinary shares value Non-vested at December 31, 2021 632,930 $ 33.54 Granted 34,700 $ 15.11 Vested (103,970) $ 52.14 Forfeited (48,800) $ 29.35 Non-vested at September 30, 2022 514,860 $ 28.94 PSUs granted in 2019 vested on the third anniversary of the grant, subject to the grantee’s continued employment. The Company granted shares to certain employees in September and December 2021 and at various dates during the nine months ended September 30, 2022 that will be earned upon achievement of defined milestones. Earned 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 December 2021 shares granted 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. ESPP During the nine months ended September 30, 2022, 9,305 ordinary shares were issued under the ESPP compared to 4,724 during the same period in 2021. As of September 30, 2022, a total of 117,997 ordinary shares remain available for issuance under the ESPP plan compared to a total of 127,302 as of September 30, 2021.

Income taxes

Income taxes	9 Months Ended
Income taxes	Sep. 30, 2022
Income taxes
Income taxes	9 Income taxes The Company recorded $0.3 and $1.3 million deferred tax benefit in relation to its operations in the United States and France during the three and nine months ended September 30, 2022, respectively. The Company recorded $0.1 million and $3.6 million in deferred income tax expense in the prior year in the United States and the Netherlands for the three and nine months ended September 30, 2021, respectively, of which nil and $3.0 million in the three and nine months periods related to the Netherlands, respectively. The effective income tax rate of -0.7% and -0.9% during the three and nine months ended September 30, 2022 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, 2021 was 0.2% and -1.1%, respectively, as the Company had recorded a valuation allowance against all 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, 2022
Basic and diluted earnings per share
Basic and diluted earnings per share	10 The potentially dilutive ordinary shares are summarized below: Three months ended September 30, Nine months ended September 30, 2022 2021 2022 2021 (in thousands, except share amounts) (in thousands, except share amounts) Numerator: Net (loss) / income attributable to ordinary shares $ (47,857) $ (36,531) $ (133,596) $ 321,381 (47,857) (36,531) (133,596) 321,381 Denominator: Weighted-average number of ordinary shares outstanding - basic 46,772,430 46,152,404 46,680,667 45,888,769 Stock options under 2014 Plans and previous plan — — — 784,942 Non-vested RSUs and PSUs — — — 105,870 Employee share purchase plan — — — 1,382 Weighted-average number of ordinary shares outstanding - diluted 46,772,430 46,152,404 46,680,667 46,780,963 Three months ended September 30, Nine months ended September 30, 2022 2021 2022 2021 Anti-dilutive ordinary share equivalents Stock options under 2014 Plans and previous plan 4,284,656 3,357,412 4,284,656 2,572,470 Non-vested RSUs and PSUs 2,328,151 841,480 2,328,151 735,610 Employee share purchase plan 721 1,219 721 1,219 Total anti-dilutive ordinary share equivalents 6,613,528 4,200,111 6,613,528 3,309,299

Subsequent events

Subsequent events	9 Months Ended
Subsequent events	Sep. 30, 2022
Subsequent events
Subsequent events	11 None.

Summary of significant accoun_2

Summary of significant accounting policies (Policies)	9 Months Ended
Summary of significant accounting policies (Policies)	Sep. 30, 2022
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 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 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, 2022, are not necessarily indicative of the results to be expected for the full year ending December 31, 2022, 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, 2021, and the notes thereto, which are included in the Company’s Annual Report Inventories The Company started producing commercial materials in April 2022 to supply CSL Behring LLC (“CSL Behring”) with etranacogene dezaparvovec (the “Product”) in accordance with the June 2020 Development and Commercial Supply Agreement between the Company and CSL Behring. From this date onwards, the Company presents the costs associated with the aforementioned activities as cost of contract manufacturing. The Company capitalizes inventory to be sold to CSL Behring to the extent it expects to generate probable future benefits from such sales. Refer to Note 3, “CSL Behring collaboration” Per ASC 330, Inventory, inventory is stated at the lower of cost or estimated net realizable value, on a first-in, first-out basis. The Company capitalizes raw materials to the extent these can be used in the manufacturing of the Product. The Company uses standard costs, approximating average costs to determine its cost basis for work in progress and finished goods. The Company’s assessment of recoverability value requires the use of estimates regarding the net realizable value of its inventory balances, including an assessment of excess or obsolete inventory. As applicable, write-downs resulting from adjustments to net realizable value will be recorded to cost of contract manufacturing.
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, 2022, as compared to the recent accounting pronouncements described in Note 2.3.23 of the Company’s Annual Report

Inventories (Tables)

Inventories (Tables)	9 Months Ended
Inventories (Tables)	Sep. 30, 2022
Inventories
Schedule of inventory balances	September 30, December 31, 2022 2021 (in thousands) Raw materials $ 1,447 $ — Work in progress 2,178 — Finished goods 450 — Inventories $ 4,075 $ —

Fair value measurement (Tables)

Fair value measurement (Tables)	9 Months Ended
Fair value measurement (Tables)	Sep. 30, 2022
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, 2021 Assets: Cash and cash equivalents $ 556,256 $ — $ — $ 556,256 Cash and cash equivalents Restricted cash 3,097 — — 3,097 Other non-current assets Total assets $ 559,353 $ — $ — $ 559,353 Liabilities: Contingent consideration — — 29,542 29,542 Contingent consideration Derivative financial instrument — — 2,805 2,805 Other non-current liabilities Consideration for post-acquisition services — — 846 846 Other non-current liabilities Total liabilities $ — $ — $ 33,193 $ 33,193 At September 30, 2022 Assets: Cash and cash equivalents $ 440,313 $ — $ — $ 440,313 Cash and cash equivalents Restricted cash 3,106 — — 3,106 Other non-current assets Total assets $ 443,419 $ — $ — $ 443,419 Liabilities: Contingent consideration — — 32,695 32,695 Current portion of contingent consideration; contingent consideration, net of current portion Derivative financial instrument — — 2,171 2,171 Other non-current liabilities Consideration for post-acquisition services — — 312 312 Other non-current liabilities Total liabilities $ — $ — $ 35,178 $ 35,178
Schedule of changes in fair value of contingent consideration	Amount of contingent consideration 2022 (in thousands) Balance at December 31, 2021 $ 29,542 Change in fair value (presented within research and development expenses) 7,510 Currency translation effects (4,357) Balance at September 30, 2022 $ 32,695

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, 2022
Accrued expenses and other current liabilities
Schedule of accrued expenses and other current liabilities	September 30, December 31, 2022 2021 (in thousands) Accruals for goods received from and services provided by vendors-not yet billed $ 13,497 $ 13,012 Personnel related accruals and liabilities 12,940 12,603 Accrued contract fulfillment costs and costs to obtain a contract — 2,872 Total $ 26,437 $ 28,487

Share-based compensation (Table

Share-based compensation (Tables)	9 Months Ended
Share-based compensation (Tables)	Sep. 30, 2022
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, 2022 2021 2022 2021 (in thousands) (in thousands) Research and development $ 3,730 $ 3,249 $ 12,090 $ 9,222 Selling, general and administrative 3,758 2,707 9,932 9,525 Cost of contract manufacturing 120 — 268 — Total $ 7,608 $ 5,956 $ 22,290 $ 18,747
Schedule of share-based compensation expense	Three months ended September 30, Nine months ended September 30, 2022 2021 2022 2021 (in thousands) (in thousands) Award type/ESPP Share options $ 3,448 $ 2,965 $ 10,141 $ 9,155 Restricted share units 3,957 2,582 11,354 8,364 Performance share units 197 403 777 1,206 ESPP 6 6 18 22 Total $ 7,608 $ 5,956 $ 22,290 $ 18,747
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 $ 27,720 2.64 Restricted share units 28,629 2.04 Total $ 56,349 2.34
Schedule of weighted-average assumptions for fair value of option issued	Three months ended September 30, Nine months ended September 30, Assumptions 2022 2021 2022 2021 Expected volatility 70% 75% 70% 75% Expected terms 10 years 10 years 10 years 10 years Risk free interest rate 4.16% 1.47% 2.12% - 4.16% 1.21 - 1.85% Expected dividend yield 0% 0% 0% 0%
Summary of RSUs activity	RSU Weighted average Number of grant-date fair ordinary shares value Non-vested at December 31, 2021 710,617 $ 38.89 Granted 1,562,133 $ 15.91 Vested (283,349) $ 39.47 Forfeited (176,110) $ 23.76 Non-vested at September 30, 2022 1,813,291 $ 20.47 Total weighted average grant date fair value of RSUs granted during the period (in $ millions) $ 24.8
Summary of PSUs activity	PSU Weighted average Number of grant-date fair ordinary shares value Non-vested at December 31, 2021 632,930 $ 33.54 Granted 34,700 $ 15.11 Vested (103,970) $ 52.14 Forfeited (48,800) $ 29.35 Non-vested at September 30, 2022 514,860 $ 28.94
2014 Plan.
Share-based compensation
Summary of option activity	Options Number of Weighted average ordinary shares exercise price Outstanding at December 31, 2021 3,308,325 $ 31.02 Granted 1,399,466 $ 15.75 Forfeited (183,870) $ 38.74 Expired (133,381) $ 35.79 Exercised (115,884) $ 6.03 Outstanding at September 30, 2022 4,274,656 $ 26.22 Thereof, fully vested and exercisable on September 30, 2022 2,068,810 $ 28.33 Thereof, outstanding and expected to vest after September 30, 2022 2,205,846 $ 24.24 Total weighted average grant date fair value of options issued during the period (in $ millions) $ 12.5 Proceeds from option sales during the period (in $ millions) $ 0.7

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, 2022
Basic and diluted earnings per share
Schedule of basic and diluted earnings per share	Three months ended September 30, Nine months ended September 30, 2022 2021 2022 2021 (in thousands, except share amounts) (in thousands, except share amounts) Numerator: Net (loss) / income attributable to ordinary shares $ (47,857) $ (36,531) $ (133,596) $ 321,381 (47,857) (36,531) (133,596) 321,381 Denominator: Weighted-average number of ordinary shares outstanding - basic 46,772,430 46,152,404 46,680,667 45,888,769 Stock options under 2014 Plans and previous plan — — — 784,942 Non-vested RSUs and PSUs — — — 105,870 Employee share purchase plan — — — 1,382 Weighted-average number of ordinary shares outstanding - diluted 46,772,430 46,152,404 46,680,667 46,780,963
Schedule of potential dilutive common shares	Three months ended September 30, Nine months ended September 30, 2022 2021 2022 2021 Anti-dilutive ordinary share equivalents Stock options under 2014 Plans and previous plan 4,284,656 3,357,412 4,284,656 2,572,470 Non-vested RSUs and PSUs 2,328,151 841,480 2,328,151 735,610 Employee share purchase plan 721 1,219 721 1,219 Total anti-dilutive ordinary share equivalents 6,613,528 4,200,111 6,613,528 3,309,299

CSL Behring collaboration (Deta

CSL Behring collaboration (Details) - USD ($) $ in Thousands		3 Months Ended		9 Months Ended
	Jun. 24, 2020	Sep. 30, 2022	Sep. 30, 2021	Sep. 30, 2022	Sep. 30, 2021	Apr. 30, 2022	Mar. 31, 2022	Dec. 31, 2021
Collaboration arrangements
Revenue		$ 1,449	$ 1,989	$ 3,738	$ 466,311
CSL Behring collaboration.
Collaboration arrangements
Upfront payment recorded	$ 450,000
Other payments received	$ 12,400
Accounts receivable		3,000		3,000				$ 2,900
Contract asset						$ 55,000	$ 55,000	$ 55,000
Collaborative revenue \| CSL Behring collaboration.
Collaboration arrangements
Revenue		700	500	2,100	900
License revenue \| CSL Behring collaboration.
Collaboration arrangements
Revenue		$ 0	$ 0	$ 0	$ 462,400

Inventories (Details)

Inventories (Details) $ in Thousands	Sep. 30, 2022 USD ($)
Inventories
Raw materials	$ 1,447
Work in progress	2,178
Finished goods	450
Inventories	$ 4,075

Fair value measurement - Assets

Fair value measurement - Assets and liabilities measured on a recurring basis (Details) - USD ($) $ in Thousands	Sep. 30, 2022	Dec. 31, 2021	Sep. 30, 2021
Assets:
Restricted cash	$ 3,106		$ 2,806
Recurring Fair Value Measurements
Assets:
Cash and cash equivalents	440,313	$ 556,256
Restricted cash	3,106	3,097
Total assets	443,419	559,353
Liabilities:
Contingent consideration	32,695	29,542
Derivative financial instrument	2,171	2,805
Consideration for post acquisition services	312	846
Total liabilities	35,178	33,193
Recurring Fair Value Measurements \| Fair value hierarchy Level 1
Assets:
Cash and cash equivalents	440,313	556,256
Restricted cash	3,106	3,097
Total assets	443,419	559,353
Recurring Fair Value Measurements \| Fair value hierarchy Level 3
Liabilities:
Contingent consideration	32,695	29,542
Derivative financial instrument	2,171	2,805
Consideration for post acquisition services	312	846
Total liabilities	$ 35,178	$ 33,193

Fair value measurement - Contin

Fair value measurement - Contingent Consideration (Details) $ in Thousands, € in Millions	9 Months Ended	12 Months Ended
	Sep. 30, 2022 USD ($)	Dec. 31, 2021 USD ($)	Sep. 30, 2022 EUR (€)
Fair Value, Liabilities Measured on Recurring Basis, Unobservable Input Reconciliation [Line Items]
Contingent consideration, net of current portion	$ 9,158	$ 29,542
Contingent consideration	32,700
Current portion of contingent consideration	$ 23,537
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%
Change in fair value (presented within research and development expenses)	$ 45,400
Acquisition of Corlieve
Fair Value, Liabilities Measured on Recurring Basis, Unobservable Input Reconciliation [Line Items]
Contingent consideration, net of current portion	$ 32,695	$ 29,542
Percentage of increase in Probability of Success	66%	55%
Change in fair value (presented within research and development expenses)	$ 7,510
Currency translation effects	(4,357)
Acquisition of Corlieve \| Maximum
Fair Value, Liabilities Measured on Recurring Basis, Unobservable Input Reconciliation [Line Items]
Contingent consideration payable upon achievement of contractually defined milestones	$ 175,200		€ 178.8
Contingent consideration discount rate percentage	13.30%	11.90%
Acquisition of Corlieve \| Minimum
Fair Value, Liabilities Measured on Recurring Basis, Unobservable Input Reconciliation [Line Items]
Contingent consideration discount rate percentage	11.90%	10.90%

Fair value measurement - BMS wa

Fair value measurement - BMS warrants (Details) $ in Millions		3 Months Ended		9 Months Ended
	Dec. 01, 2020 USD ($) item	Sep. 30, 2022 USD ($)	Sep. 30, 2021 USD ($)	Sep. 30, 2022 USD ($)	Sep. 30, 2021 USD ($)	Dec. 31, 2021 USD ($)
Collaboration arrangements
Number of Collaboration Targets \| item	4
Collaboration Agreement, Cash Payable	$ 70
Derivative gain (loss) on derivative net		$ 0	$ 0	$ 0.6	$ 0
Derivative, Gain (Loss), Statement of Income or Comprehensive Income [Extensible Enumeration]				Other Nonoperating Income (Expense).
Non-current liabilities
Collaboration arrangements
Fair value of the derivative financial liability		$ 2.2		$ 2.2		$ 2.8
Collaboration Agreement, If 70 Million Is Greater Than Five Percent Of Net Proceeds \| Derecognition Of Warrants
Collaboration arrangements
Threshold Percentage Of Net Proceeds	5
Percentage Of Net Proceeds Payable	5
Collaboration Agreement, If 70 Million Is Lesser Than One Percent Of Net Proceeds \| Derecognition Of Warrants
Collaboration arrangements
Threshold Percentage Of Net Proceeds	1
Percentage Of Net Proceeds Payable	1

Accrued expenses and other cu_3

Accrued expenses and other current liabilities (Details) - USD ($) $ in Thousands	Sep. 30, 2022	Dec. 31, 2021
Accrued expenses and other current liabilities
Accruals for goods received from and services provided by vendors-not yet billed	$ 13,497	$ 13,012
Personnel related accruals and liabilities	12,940	12,603
Accrued contract fulfillment costs and costs to obtain a contract		2,872
Total	$ 26,437	$ 28,487

Long-term debt (Details)

Long-term debt (Details) - USD ($) $ in Millions				3 Months Ended		9 Months Ended
Long-term debt (Details) - USD ($) $ in Millions	Apr. 01, 2024	Apr. 01, 2023	Jan. 29, 2021	Sep. 30, 2022	Sep. 30, 2021	Sep. 30, 2022	Sep. 30, 2021	Dec. 31, 2021
2021 Amended Facility
Long-term Debt
Proceed from drew down			$ 35
2018 and 2021 Amended Facility
Long-term Debt
Foreign currency loss				$ 6.6	$ 1.8	$ 15	$ 4
Interest expense recorded					$ 1.9		$ 5.2
Restated Facility 2021
Long-term Debt
Outstanding debt				$ 100		100
Proceed from drew down						$ 30
Interest rate (as a percent)				7.95%		7.95%
Back-end fee (as a percent)						4.85%
Back-end fees due				$ 2.5		$ 2.5
Amortized cost net of discount and debt issuance costs				103.3		103.3		$ 101.6
Interest expense recorded				3		8.1
Restated Facility 2021 \| Tranche One
Long-term Debt
Outstanding debt				$ 70		$ 70
Restated Facility 2021 \| Prime Rate
Long-term Debt
Variable interest rate basis						7.95%
Discount rate (as a percent)				3.25%		3.25%
Venture debt loan facility \| Restated Facility 2021
Long-term Debt
Minimum cash and cash equivalents in U.S. bank accounts						65%	100%
Assets pledged to secure facilities by directly or indirectly						$ 638.2
Assets not being pledged to secure facilities by directly or indirectly						75.3
Venture debt loan facility \| Restated Facility 2021 \| Corlieve Therapeutics SAS
Long-term Debt
Assets not being pledged to secure facilities by directly or indirectly						$ 80.2
Subsequent events. \| Venture debt loan facility \| Restated Facility 2021
Long-term Debt
Minimum cash and cash equivalents in U.S. bank accounts	30%	50%

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
	May 31, 2021	Jun. 30, 2021	Jun. 30, 2018	Sep. 30, 2022	Sep. 30, 2021	Sep. 30, 2022	Sep. 30, 2021
2014 Plan
Share-based compensation
Increase in authorized shares	8,601,471	12,601,471
Share-based compensation expense				$ 7,608	$ 5,956	$ 22,290	$ 18,747
Unrecognized compensation costs				56,349		$ 56,349
Weighted-average remaining period for recognition (in years)						2 years 4 months 2 days
ESPP
Share-based compensation
Share-based compensation expense				6	6	$ 18	22
2014 Plans and ESPP
Share-based compensation
Share-based compensation expense				7,608	5,956	22,290	18,747
Research and development expenses \| 2014 Plans and ESPP
Share-based compensation
Share-based compensation expense				3,730	3,249	12,090	9,222
Selling, general and administrative expense \| 2014 Plans and ESPP
Share-based compensation
Share-based compensation expense				3,758	2,707	9,932	9,525
Cost of manufacturing services revenues \| 2014 Plans and ESPP
Share-based compensation
Share-based compensation expense				120		268
Share options \| 2014 Plan
Share-based compensation
Unrecognized compensation costs				27,720		$ 27,720
Weighted-average remaining period for recognition (in years)						2 years 7 months 20 days
Share options \| 2014 Plans and ESPP
Share-based compensation
Share-based compensation expense				3,448	2,965	$ 10,141	9,155
Restricted share units ("RSUs") \| 2014 Plan
Share-based compensation
Unrecognized compensation costs				28,629		$ 28,629
Weighted-average remaining period for recognition (in years)						2 years 14 days
Restricted share units ("RSUs") \| 2014 Plans and ESPP
Share-based compensation
Share-based compensation expense				3,957	2,582	$ 11,354	8,364
Performance Share Awards \| 2014 Plans and ESPP
Share-based compensation
Share-based compensation expense				$ 197	$ 403	$ 777	$ 1,206
Employee Stock \| ESPP
Share-based compensation
Ordinary shares available for issue			150,000	117,997	127,302	117,997	127,302
Discounted rate for purchase of shares			85%

Share-based compensation - Opti

Share-based compensation - Option activity and weighted-average assumptions (Details) - USD ($) $ / shares in Units, $ in Millions	3 Months Ended		9 Months Ended
	Sep. 30, 2022	Sep. 30, 2021	Sep. 30, 2022	Sep. 30, 2021
Weighted average exercise price
Total weighted average grant date fair value of options issued during the period (in $ millions)			$ 12.5
Proceeds from option sales during the period (in $ millions)			$ 0.7
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)			3,308,325
Granted (in shares)			1,399,466
Forfeited (in shares)			(183,870)
Expired (in shares)			(133,381)
Exercised (in shares)			(115,884)
Outstanding at end of the period (in shares)	4,274,656		4,274,656
Thereof, fully vested and exercisable at end of period (in shares)	2,068,810		2,068,810
Thereof, outstanding and expected to vest at end of period (in shares)	2,205,846		2,205,846
Weighted average exercise price
Outstanding at beginning of the period (in dollars per share)			$ 31.02
Granted (in dollars per share)			15.75
Forfeited (in dollars per share)			38.74
Expired (in dollars per share)			35.79
Exercised (in dollars per share)			6.03
Outstanding at end of period (in dollars per share)	$ 26.22		26.22
Thereof, fully vested and exercisable at end of period (in dollars per share)	$ 28.33		28.33
Outstanding and expected to vest at end of the period (in dollars per share)			$ 24.24
Weighted-average assumptions used to estimate fair value of share options granted during year
Expected volatility (as a percent)	70%	75%	70%	75%
Expected terms (in years)	10 years	10 years	10 years	10 years
Risk free interest rate, minimum (as a percent)	4.16%	1.47%	2.12%	1.21%
Risk free interest rate, maximum (as a percent)			4.16%	1.85%
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, 2022 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	710,617
Granted (in shares) \| shares	1,562,133
Vested (in shares) \| shares	(283,349)
Forfeited (in shares) \| shares	(176,110)
Non-vested at end of period (in shares) \| shares	1,813,291
Weighted average grant-date fair value
Non-vested at beginning of period (in dollars per share) \| $ / shares	$ 38.89
Granted (in dollars per share) \| $ / shares	15.91
Vested (in dollars per share) \| $ / shares	39.47
Forfeited (in dollars per share) \| $ / shares	23.76
Non-vested at end of period (in dollars per share) \| $ / shares	$ 20.47
Other disclosure
Total weighted average grant date fair value of RSUs granted during the period (in millions) \| $	$ 24.8
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) - $ / shares	1 Months Ended		9 Months Ended
	Dec. 31, 2021	Sep. 30, 2021	Sep. 30, 2022
Other disclosure
Service period	3 years	1 year
Performance Share Awards \| 2014 Plan
Number of shares
Non-vested at beginning of period (in shares)			632,930
Granted (in shares)			34,700
Vested (in shares)			(103,970)
Forfeited (in shares)			(48,800)
Non-vested at end of period (in shares)	632,930		514,860
Weighted average grant-date fair value
Non-vested at beginning of period (in dollars per share)			$ 33.54
Granted (in dollars per share)			15.11
Vested (in dollars per share)			52.14
Forfeited (in dollars per share)			29.35
Non-vested at end of period (in dollars per share)	$ 33.54		$ 28.94

Share-based compensation - Empl

Share-based compensation - Employee Share Purchase Plan (Details) - ESPP - Employee Stock - shares	9 Months Ended
	Sep. 30, 2022	Sep. 30, 2021	Jun. 30, 2018
Share-based compensation
Number of shares issued	9,305	4,724
Ordinary shares available for issue	117,997	127,302	150,000

Income taxes - Effective Tax Ra

Income taxes - Effective Tax Rate (Details) - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2022	Sep. 30, 2021	Sep. 30, 2022	Sep. 30, 2021
Effective income tax rate	(0.70%)	0.20%	(0.90%)	(1.10%)
Enacted tax rate	25.80%		25.80%
Deferred tax (benefit) expense			$ (1,263)	$ 3,560
United States and France
Deferred tax (benefit) expense	$ (300)		$ (1,300)
United States and Netherland
Deferred tax (benefit) expense		$ 100		3,600
Netherland
Deferred tax (benefit) expense		$ 0		$ 3,000

Basic and diluted earnings pe_3

Basic and diluted earnings per share - Ordinary share (Details) - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2022	Sep. 30, 2021	Sep. 30, 2022	Sep. 30, 2021
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Net (loss) / income attributable to ordinary shares	$ (47,857)	$ (36,531)	$ (133,596)	$ 321,381
Weighted-average number of ordinary shares outstanding - basic	46,772,430	46,152,404	46,680,667	45,888,769
Weighted-average number of ordinary shares outstanding - diluted	46,772,430	46,152,404	46,680,667	46,780,963
ESPP
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Share based payment arrangements				1,382
Stock options \| Previous plan
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Share based payment arrangements				784,942
Non-vested RSUs and PSUs
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Share based payment arrangements				105,870

Basic and diluted earnings pe_4

Basic and diluted earnings per share (Details) - shares	3 Months Ended		9 Months Ended
Basic and diluted earnings per share (Details) - shares	Sep. 30, 2022	Sep. 30, 2021	Sep. 30, 2022	Sep. 30, 2021
Basic and diluted earnings per share
Total potential dilutive ordinary shares	6,613,528	4,200,111	6,613,528	3,309,299
ESPP
Basic and diluted earnings per share
Total potential dilutive ordinary shares	721	1,219	721	1,219
Stock options
Basic and diluted earnings per share
Total potential dilutive ordinary shares	4,284,656	3,357,412	4,284,656	2,572,470
Non-vested RSUs and PSUs
Basic and diluted earnings per share
Total potential dilutive ordinary shares	2,328,151	841,480	2,328,151	735,610