What changed in this filing

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Item 9A. Controls and Procedures

2022-02-25 compared with 2021-03-01 · 554 added, 411 removed, 896 unchanged (52% of the section changed)

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Evaluation of Disclosure Controls and Procedures

Our management, with the participation of our chief executive officer (“CEO”), who also serves as our chief financial officer, evaluated the effectiveness of our disclosure controls and procedures (as defined in Rules 13a-15(e) and 15d-15(e) under the Securities Exchange Act of 1934, as amended (the “Exchange Act”)) as of December 31, 2020.

Based on such evaluation, our CEO has concluded that as of December 31, 2020, our disclosure controls and procedures were effective.

Our management, with the participation of our chief executive officer (“CEO”, our principal executive officer) and chief financial officer (“CFO”, our principal financial officer), evaluated the effectiveness of our disclosure controls and procedures (as defined in Rules 13a-15(e) and 15d-15(e) under the Securities Exchange Act of 1934, as amended (the “Exchange Act”)) as of December 31, 2021.

Based on such evaluation, our CEO and CFO have concluded that as of December 31, 2021, our disclosure controls and procedures were effective.

Management’s Annual Report on Internal Control Over Financial Reporting

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We assessed the effectiveness of our internal control over financial reporting as of December 31, 2021.

This assessment was performed under the direction and supervision of our CEO and based on criteria established in Internal Control—Integrated Framework (2013) issued by the Committee of Sponsoring Organizations of the Treadway Commission (“COSO”).

This assessment was performed under the direction and supervision of our CEO and CFO and based on criteria established in Internal Control - Integrated Framework (2013) issued by the Committee of Sponsoring Organizations of the Treadway Commission (“COSO”).

Our management’s assessment of the effectiveness of our internal control over financial reporting included testing and evaluating the design and operating effectiveness of our internal controls.

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Inherent Limitations of Internal Controls

Our management, including our CEO, does not expect that our disclosure controls and procedures or our internal controls will prevent all errors and all fraud.

Our management, including our CEO and CFO, does not expect that our disclosure controls and procedures or our internal controls will prevent all errors and all fraud.

A control system, no matter how well conceived and operated, can provide only reasonable, not absolute, assurance that the objectives of the control system are met.

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During the fourth quarter of 2021, there were no changes in our internal control over financial reporting (as defined in Rule 13a-15(f) under the Exchange Act) that materially affected, or are reasonably likely to materially affect, our internal control over financial reporting.

We have not experienced any material impact to our internal controls over financial reporting even though a large group of our employees are working remotely due to the COVID-19 pandemic.

We are continually monitoring and assessing the impact COVID-19 has on the operating effectiveness of our internal controls.

Other Information

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Report of Independent Registered Public Accounting Firm – KPMG Accountants N.V.

Report of Independent Registered Public Accounting Firm – PricewaterhouseCoopers Accountants N.V.

Consolidated Balance Sheets as of December 31, 2021 and 2020

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Report of Independent Registered Public Accounting Firm - KPMG Accountants N.V.

Report of Independent Registered Public Accounting Firm – PricewaterhouseCoopers Accountants N.V.

, Amstelveen, The Netherlands (PCAOB ID 1012 )

Consolidated Balance Sheets as of December 31, 2021 and 2020

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We have audited the accompanying consolidated balance sheets of uniQure N.V.

and subsidiaries (the Company) as of December 31, 2020 and 2019, the related consolidated statements of operations and comprehensive loss, shareholders’ equity, and cash flows for the years then ended, and the related notes (collectively, the consolidated financial statements).

and subsidiaries (‘the Company’) as of December 31, 2021 and 2020, the related consolidated statement of operations and comprehensive loss, shareholders’ equity, and cash flows for each of the years in the three-year period ended December 31, 2021, and the related notes (collectively, the consolidated financial statements).

We also have audited the Company’s internal control over financial reporting as of December 31, 2021, based on criteria established in Internal Control – Integrated Framework (2013) issued by the Committee of Sponsoring Organizations of the Treadway Commission.

In our opinion, the consolidated financial statements referred to above present fairly, in all material respects, the financial position of the Company as of December 31, 2020 and 2019, and the results of its operations and its cash flows for the years then ended, in conformity with U.S.

In our opinion, the consolidated financial statements referred to above present fairly, in all material respects, the financial position of the Company as of December 31, 2021 and 2020, and the results of its operations and its cash flows for each of the years in the three year-period ended December 31, 2021, in conformity with U.S.

generally accepted accounting principles.

Also in our opinion, the Company maintained, in all material respects, effective internal control over financial reporting as of December 31, 2021 based on criteria established in Internal Control – Integrated Framework (2013) issued by the Committee of Sponsoring Organizations of the Treadway Commission.

Change in Accounting Principle

As discussed in Note 6, to the consolidated financial statements, the Company changed its method of accounting for leases as of January 1, 2019 due to the adoption of ASC 842, Leases .

Basis for Opinions

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Also, projections of any evaluation of effectiveness to future periods are subject to the risk that controls may become inadequate because of changes in conditions, or that the degree of compliance with the policies or procedures may deteriorate.

Critical Audit Matters

Critical Audit Matter

The critical audit matter communicated below is a matter arising from the current period audit of the consolidated financial statements that was communicated or required to be communicated to the audit committee and that:

(1) relates to accounts or disclosures that are material to the consolidated financial statements and (2) involved our especially challenging, subjective, or complex judgments.

(1) relates to accounts or disclosures that are material to the consolidated f inancial statements and (2) involved our especially challenging, subjective, or complex judgments.

The communication of a critical audit matter does not alter in any way our opinion on the consolidated financial statements, taken as a whole, and we are not, by communicating the critical audit matter below, providing a separate opinion on the critical audit matter or on the accounts or disclosures to which it relates.

Assessment of the satisfaction of the license revenue performance obligation within the BMS collaboration and license agreement

As discussed in Notes 2.3.18 and 3 to the consolidated financial statements, uniQure biopharma B.V., a subsidiary of uniQure N.V.

(the Company) entered into an amended collaboration and license agreement (CLA) with Bristol-Myers Squibb Company (BMS) on December 1, 2020.

Based on the terms of the amended agreement, the Company determined that it materially satisfied its performance obligation in relation to the license revenue as of December 1, 2020, and as such recorded the residual balance of unrecognized license revenue of $27.8 million in profit and loss as license revenues from related party.

We identified the assessment of the satisfaction of the license revenue performance obligation as a critical audit matter.

A high degree of subjective, complex auditor judgement was required in assessing if the remaining rights of the license revenue performance obligation were materially satisfied as of December 1, 2020.

Evaluation of the acquisition of Corlieve as a business combination

As discussed in Notes 2.3.5 and 3 to the consolidated financial statements, the Company acquired 97.7% of the outstanding ordinary shares of Corlieve for a total purchase price of EUR 65.8 million.

The Company applied the applicable accounting guidance which requires the acquirer to assess whether the acquisition should be accounted for as an asset acquisition or a business combination.

We identified the evaluation of the acquisition as a business combination to be a critical audit matter.

This evaluation required significant auditor judgement to assess whether or not substantially all of the fair value of the gross assets acquired is concentrated in a single identifiable asset or group of similar identifiable assets based on the similarities of risks associated with managing and creating outputs from the assets, including the acquired workforce and identification of intangible assets.

The following are the primary procedures we performed to address this critical audit matter:

- We evaluated the design and tested the operating effectiveness of an internal control related to the evaluation of the performance obligation and when it was materially satisfied.

- In order to assess that the remaining rights of the license revenue performance obligation were materially satisfied, we examined the amended collaboration and license agreement between the Company and BMS.

- In order to assess the satisfaction of the performance obligation, we examined minutes of joint steering committee meetings between the Company and BMS, evaluated the services delivered and performed interviews with the Company’s finance and business operations department.

‒ We evaluated the design and tested the operating effectiveness of certain internal controls related to the business combination process, including controls related to technical accounting review and over the completeness of assets and liabilities acquired.

‒ We read the sale and purchase agreement and performed inquires with management and business development personnel to understand the business rationale for acquiring Corlieve.

‒ We read the employment terms and inquired of management and business development personnel to challenge management’s judgement that the employees acquired represent an organized workforce.

‒ We involved valuation professionals with specialized skills and knowledge, who assisted us in evaluating the Company’s identification of intangible assets acquired.

/s/ KPMG Accountants N.V.

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Amstelveen , The Netherlands

March 1, 2021

Report of Independent Registered Public Accounting Firm

To the Management Board and Shareholders of uniQure N.V.:

Opinion on the Financial Statements

We have audited the consolidated statements of operations and comprehensive loss, of shareholders’ equity and of cash flows of uniQure N.V.

and its subsidiaries (the “Company”) for the year ended December 31, 2018, including the related notes (collectively referred to as the “consolidated financial statements”).

In our opinion, the consolidated financial statements present fairly, in all material respects, the results of operations and cash flows of the Company for the year ended December 31, 2018 in conformity with accounting principles generally accepted in the United States of America.

Basis for Opinion

These consolidated financial statements are the responsibility of the Company's management.

Our responsibility is to express an opinion on the Company’s consolidated financial statements based on our audit.

We are a public accounting firm registered with the Public Company Accounting Oversight Board (United States) (PCAOB) and are required to be independent with respect to the Company in accordance with the U.S.

federal securities laws and the applicable rules and regulations of the Securities and Exchange Commission and the PCAOB.

We conducted our audit of these consolidated financial statements in accordance with the standards of the PCAOB.

Those standards require that we plan and perform the audit to obtain reasonable assurance about whether the consolidated financial statements are free of material misstatement, whether due to error or fraud.

Our audit included performing procedures to assess the risks of material misstatement of the consolidated financial statements, whether due to error or fraud, and performing procedures that respond to those risks.

Such procedures included examining, on a test basis, evidence regarding the amounts and disclosures in the consolidated financial statements.

Our audit also included evaluating the accounting principles used and significant estimates made by management, as well as evaluating the overall presentation of the consolidated financial statements.

We believe that our audit provides a reasonable basis for our opinion.

PricewaterhouseCoopers Accountants N.V.

Amsterdam, the Netherlands

February 25, 2022

We served as the Company's auditor from 2006 to 2019.

CONSOLIDATED BALANCE SHEET S

CONSOLIDATED BALANCE SHEETS

(in thousands, except share and per share amounts)

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Cash and cash equivalents

Accounts receivables

Accounts receivable from related party

Accounts receivable and contract asset

Prepaid expenses

Other current assets

Other current assets and receivables

Total current assets

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Intangible assets, net

Restricted cash

Deferred tax asset

Deferred tax assets, net

Other non-current assets

Total non-current assets

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Current portion of operating lease liabilities

Current portion of deferred revenue

Total current liabilities

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Operating lease liabilities, net of current portion

Deferred revenue, net of current portion

Derivative financial instruments related party

Contingent consideration

Deferred tax liability, net

Other non-current liabilities

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Ordinary shares, € 0.05 par value:

60,000,000 shares authorized at December 31, 2020 and December 31, 2019 and 44,777,799 and 43,711,954 ordinary shares issued and outstanding at December 31, 2020 and December 31, 2019, respectively

80,000,000 shares authorized as of December 31, 2021 and 60,000,000 shares authorized as of December 31, 2020 and 46,298,635 and 44,777,799 ordinary shares issued and outstanding as of December 31, 2021 and December 31, 2020, respectively

Additional paid-in-capital

Accumulated other comprehensive income / (loss)

Accumulated other comprehensive (loss) / income

Accumulated deficit

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Operating expenses:

Cost of contract revenues

Research and development expenses

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Other expense

Loss from operations

Income / (loss) from operations

Interest income

Interest expense

Foreign currency (losses) / gains, net

Other non-operating gains / (losses), net

Loss before income tax income / (expense)

Income tax income / (expense)

Other comprehensive income / (loss):

Foreign currency translation adjustments net of tax impact of nil for the year ended December 31, 2020 (2019:

nil and 2018:

$( 0.2 ) million)

Total comprehensive loss

Basic and diluted net loss per ordinary share

Weighted average shares used in computing basic and diluted net loss per ordinary share

Foreign currency gains / (losses), net

Other non-operating (losses) / gains, net

Income / (loss) before income tax (expense) / benefit

Income tax (expense) / benefit

Net income / (loss)

Other comprehensive (loss) / gain:

Foreign currency translation adjustments

Total comprehensive gain / (loss)

Earnings per ordinary share - basic

Basic net income / (loss) per ordinary share

Earnings per ordinary share - diluted

Diluted net income / (loss) per ordinary share

Weighted average shares - basic

Weighted average shares - diluted

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

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shareholders’

(loss)/income

(in thousands, except share data)

Balance at December 31, 2018

Cumulative effect of retroactive implementation of ASC 606 Revenue recognition

Loss for the period

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Follow-on public offering

Hercules warrants exercise

Exercises of share options

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Other comprehensive income

Follow-on public offering

Hercules warrants exercise

Exercise of share options

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Balance at December 31, 2020

Loss for the period

Other comprehensive income

Income for the period

Other comprehensive loss

Issuance of ordinary shares

Income tax benefit of past share issuance cost

Exercise of share options

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Cash flows from operating activities

Adjustments to reconcile net loss to net cash used in operating activities:

Depreciation, amortization, and impairment losses

Net income / (loss)

Adjustments to reconcile net income / (loss) to net cash generated from / (used in) operating activities:

Depreciation and amortization expense

Share-based compensation expense

Change in fair value of derivative financial instruments and contingent consideration

Unrealized foreign exchange losses / (gains)

Deferred tax (income) / expense

Change in lease incentives

Deferred tax expense / (income)

Changes in fair value of contingent consideration and derivative financial instruments

Unrealized foreign exchange (gains) / losses, net

Change in deferred revenue

Other non-cash items, net

Changes in operating assets and liabilities:

Accounts receivable and accrued income, prepaid expenses, and other current assets

Contract asset related to CSL Behring milestone payments

Accounts receivable, prepaid expenses, and other current assets and receivables

Accounts payable

Accrued expenses, other liabilities, and operating leases

Net cash used in operating activities

Net cash generated from / (used in) operating activities

Cash flows from investing activities

Acquisition of Corlieve, net of cash acquired

Purchases of intangible assets

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Cash flows from financing activities

Proceeds from issuance of shares related to employee stock option and purchase plans

Proceeds from loan increment, net of debt issuance costs

Proceeds from public offering of shares, net of issuance costs

Proceeds from loan increment

Proceeds from issuance of ordinary shares

Proceeds from issuance of ordinary shares related to employee stock option and purchase plans

Repayment of debt acquired through acquisition of Corlieve

Share issuance costs from issuance of ordinary shares

Proceeds from exercise of warrants

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Currency effect on cash, cash equivalents and restricted cash

Net (decrease) / increase in cash, cash equivalents and restricted cash

Net increase / (decrease) in cash, cash equivalents and restricted cash

Cash, cash equivalents and restricted cash at beginning of period

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Cash paid for interest

Non-cash increase / (decrease) in accounts payables and accrued expenses and other current liabilities related to purchases of intangible assets and property, plant, and equipment

Non-cash increase in accounts payables and accrued expenses and other current liabilities related to purchases of property, plant, and equipment

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

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to uniQure N.V.

The Company is registered in the trade register of the Dutch Chamber of Commerce (Kamer van Koophandel) under number 54385229.

The Company’s headquarters are in Amsterdam, the Netherlands, and its registered office is located at Paasheuvelweg 25a, Amsterdam 1105 BP, the Netherlands and its telephone number is +31 20 240 6000.

The Company is registered in the trade register of the Dutch Chamber of Commerce ( Kamer van Koophandel ) in Amsterdam, the Netherlands under number 54385229.

The Company’s headquarters are in Amsterdam, the Netherlands, and its registered office is located at Paasheuvelweg 25, Amsterdam 1105 BP, the Netherlands and its telephone number is +31 20 240 6000.

The Company’s website address is www.uniqure.com.

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

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

Summary of significant accounting policies

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GAAP and Securities and Exchange Commission (“SEC”) rules and regulations, 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 consolidated financial statements and reported amounts of revenues and expenses during the reporting period.

Estimates and assumptions are primarily made in relation to the treatment of the commercialization and license agreement entered into between the Company and CSL Behring LLC (“CSL Behring Agreement”), the December 1, 2020, amendment (“amended BMS CLA”) of the 2015 collaboration and license agreement (“BMS CLA”) between the Company and Bristol-Myers Squibb (“BMS”), share-based payments, valuation allowances for deferred tax assets, and accounting for operating leases under ASC 842.

Estimates and assumptions are primarily made in relation to the treatment of the share and purchase agreement (“SPA”) entered into on June 21, 2021 to acquire all of the outstanding ordinary shares of Corlieve Therapeutics SAS (“Corlieve”), a privately held French gene therapy company (“Corlieve Transaction”), the treatment of the commercialization and license agreement entered into (“CSL Behring Agreement”) between the Company and CSL Behring LLC (“CSL Behring”), the assessment of a valuation allowance on the Company’s deferred tax assets in the Netherlands and the U.S., and the December 1, 2020, amendment (“amended BMS CLA”) of the 2015 collaboration and license agreement (“BMS CLA”) between the Company and Bristol-Myers Squibb (“BMS”).

If actual results differ from the Company’s estimates, or to the extent these estimates are adjusted in future periods, the Company’s results of operations could either benefit from, or be adversely affected by, any such change in estimate.

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2.3.3 Foreign currency translation

The functional currency of the Company and each of its entities (except for uniQure Inc.) is the euro (€).

The functional currency of the Company and each of its entities (except for uniQure Inc.

and Corlieve AG) is the euro (€).

This represents the currency of the primary economic environment in which the entities operate.

The functional currency of uniQure Inc.

dollar ($) and the functional currency of Corlieve AG is the Swiss Franc.

The consolidated financial statements are presented in U.S.

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The carrying amount of cash and cash equivalents, accounts receivable from collaborators, 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.

2.3.5 Corlieve transaction

On July 30, 2021 (“Acquisition Date”), the Company acquired Corlieve.

The Company evaluated the Corlieve transaction as to whether or not the transaction should be accounted for as a business combination or asset acquisition.

Refer to Note 3 “ Corlieve transaction ” for further detail.

Goodwill represents the excess of the fair value of the consideration transferred over the fair value of the net assets assumed in a business combination.

Goodwill is not amortized but is evaluated for impairment on an annual basis and between annual tests if we become aware of any events occurring or changes in circumstances that would more likely than not reduce the fair value of the reporting unit below its carrying amount.

As of December 31, 2021, the Company has not recognized any impairment charges related to goodwill.

Refer to Note 3 “ Corlieve transaction ” for further detail.

Acquired research and development

The Company identified various licenses that combined with the results of the research and development activities conducted in relation to AMT-260 since incorporation of Corlieve in 2019 constitute an In-process research and development intangible asset (“IPR&D Intangible Asset”).

The IPR&D Intangible Asset is considered to be indefinite-lived until the completion or abandonment of the associated research and development efforts and is not amortized.

If and when development is completed, which generally occurs when regulatory approval to market a product is obtained, the associated asset would be deemed finite-lived and would then be amortized based on its respective useful life at that point in time.

As of December 31, 2021, the Company has not recognized any impairment charges related to the IPR&D Intangible Asset.

In case of abandonment, the IPR&D Intangible Asset will be written-off.

In accordance with ASC 350, Intangibles – Goodwill and Other, the Company tests indefinite-lived intangible assets for impairment on an annual basis and between annual tests if the Company becomes aware of any events occurring or changes in circumstances that would indicate the fair value of the IPR&D Intangible Asset is below its carrying amount.

Refer to Note 3 “ Corlieve transaction ” for further detail.

Contingent consideration

Each reporting period, the Company revalues the contingent consideration obligations associated with the Corlieve transaction to their fair value and records changes in the fair value within research and development expenses.

Changes in contingent consideration result from changes in assumptions regarding the probabilities of achieving the relevant milestones, or probability of success (“POS”), the estimated timing of achieving such milestones, and the interest rate to discount the payments.

Payments made soon after the acquisition date are recorded as cash flows from financing activities, and payments, or the portion of the payments, not made soon after the acquisition date are recorded as cash flows from operating activities.

Refer to Note 3 “ Corlieve transaction ” for further detail.

2.3.6 Notes to the consolidated statements of cash flows

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The Company views its operations and manages its business as one operating segment, which comprises the discovery, development, and commercialization of innovative gene therapies.

2.3.7 Net loss per share

2.3.8 Net income / (loss) per share

The Company follows the provisions of ASC 260, Earnings Per Share .

In accordance with these provisions, loss per share is calculated by dividing net loss by the weighted average number of ordinary shares outstanding during the period.

Diluted net loss per share reflects the dilution that would occur if share options or warrants to issue ordinary shares were exercised, or performance or restricted share units were distributed.

In accordance with these provisions, net income / (loss) per share is calculated by dividing net income / (loss) by the weighted average number of ordinary shares outstanding during the period.

Diluted net income / (loss) per share reflects the dilution that would occur if share options or warrants to issue ordinary shares were exercised, performance or restricted share units were distributed, or shares under the employee share purchase plan were issued.

However, potential ordinary shares are excluded if their effect is anti-dilutive.

The Company currently has no dilutive securities due to the net loss position and as such, basic and diluted net loss per share are the same for the periods presented.

Refer to Note 16 “ Basic and diluted earnings per share ” for further information.

2.3.9 Impairment of long-lived assets

Long-lived assets, which include property, plant, and equipment and finite-lived intangible assets, are reviewed for impairment whenever events or changes in circumstances indicate that the carrying value of an asset or asset group may not be recoverable.

Right-of-use assets are also reviewed for impairment in accordance with ASC 360.

Right-of-use assets are also reviewed for impairment in accordance with ASC 360, Property, Plant, and Equipment .

The recoverability of the carrying value of an asset or asset group depends on the successful execution of the Company’s business initiatives and its ability to earn sufficient returns on approved products and product candidates.

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Fair value is determined through various valuation techniques, including discounted cash flow models, quoted market values, and third-party independent appraisals, as considered necessary.

Goodwill is not amortized but is evaluated for impairment within the Company’s single reporting unit on an annual basis, during the fourth quarter, or more frequently if an event occurs or circumstances change that would more-likely-than-not reduce the fair value of the Company’s reporting unit below its carrying amount.

The Company performs the same quantitative analysis discussed above for long-lived assets and finite-lived intangible assets

Refer to Note 2.3.5 “ Corlieve transaction ” for information on impairment testing related to goodwill and acquired research and development intangible assets.

2.3.10 Property, plant, and equipment

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2.3.11 Leases

The Company adopted ASC 842 using the modified retrospective approach with an effective date as of the beginning of the Company’s fiscal year, January 1, 2019, to operating leases that existed on that date.

Comparative financial information related to profit and loss and cash flows for the twelve-month period ended December 31, 2018, was not recast under the new standard, and continues to be presented under ASC 840.

The Company adopted ASC 842, Leases using the modified retrospective approach with an effective date as of the beginning of the Company’s fiscal year, January 1, 2019, to operating leases that existed on that date.

The Company measured lease liabilities at the present value of the future lease payments as of January 1, 2019.

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Absent a lease modification, the Company will continue to utilize the January 1, 2019, incremental borrowing rate.

For leases recognized after the adoption date, the Company determines if an arrangement is a lease at inception.

Operating lease right-of-use assets and lease liabilities are initially recognized based on the present value of future minimum lease payments over the lease term at commencement date calculated using an incremental borrowing rate applicable to the lease asset, unless the implicit rate is readily available.

Lease terms may include options to extend or terminate the lease when it is reasonably certain that the Company will exercise that option.

Leases with a term of twelve months or less are not recognized on the consolidated balance sheets.

The Company recognizes lease cost on a straight-line basis and presents these costs as operating expenses within the Consolidated statements of operations and comprehensive loss.

The Company presents lease payments and landlord incentive payments within cash flows from operations within the Consolidated statements of cash flows.

The financial results for the years ended December 31, 2020 and 2019 are presented in accordance with ASC 842, while the financial results for the year ended December 31, 2018 are presented in accordance with the Company’s historical accounting policy based on ASC 840.

The Company presents lease payments within cash flows from operations within the Consolidated statements of cash flows.

2.3.12 Other (non) current assets

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2.3.14 Accounts receivable

Accounts receivables include amounts due from services provided to the Company’s collaboration partner as well as unconditional rights to consideration from its collaboration partners.

Accounts receivables include amounts due from services provided to the Company’s licensing and collaboration partners as well as unconditional rights to consideration from its licensing and collaboration partners.

2.3.15 Accounts payable and accrued expenses

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Prepaid contributions are recognized as an asset to the extent that a cash refund or a reduction in the future payments is available.

Starting in 2016, the Company adopted a qualified 401(k) Plan for all employees at its Lexington facility in the USA, which offers both a pre-tax and post-tax (Roth) component.

Employees may contribute up to 50 % of their pre-tax compensation, which is subject to IRS statutory limits for each calendar year.

In 2016, the Company adopted a qualified 401(k) Plan for all employees located in the United States.

The 401(k) Plan offers both a pre-tax and post-tax (Roth) component.

Employees may contribute up to the IRS statutory limit each calendar year.

The Company matches $ 0.50 for every $ 1.00 contributed to the plan by participants up to 6 % of base compensation.

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Forfeitures of employee options are recognized as they occur.

Compensation expense related to Performance Share Units is recognized when the Company considers achievement of the milestones to be probable.

The requirements of ASC 718 are also applied to nonemployee share-based payment transactions except for specific guidance on certain inputs to an option-pricing model and the attribution of cost.

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2.3.19 Revenue recognition

The Company primarily generates revenue from its collaboration, research, and license agreements with its collaboration partner BMS for the development and commercialization of product candidates.

The Company initially entered into these agreements in 2015 and amended them in 2020.

On January 1, 2018, the Company adopted new revenue recognition policies in accordance with ASC 606 using the modified retrospective approach.

The Company primarily generates revenue from its commercialization and license agreement with CSL Behring and its collaboration, research, and license agreements with BMS for the development and commercialization of product candidates.

CSL Behring collaboration

On June 24, 2021 (“Signing Date”), the Company entered into a commercialization and license agreement pursuant to which CSL Behring received exclusive global rights to etranacogene dezaparvovec (“Product”).

The Company concluded that CSL Behring is a customer in accordance with ASC 606, Revenue from Contracts with Customers and identified two material performance obligations related to the CSL Behring Agreement:

(i) Sale of the exclusive global rights to the Product (“License Sale”);

(ii) Generate information to support the regulatory approval of the current and next generation manufacturing process of Product and to provide any such information generated to CSL Behring (“Manufacturing Development”).

These performance obligations are considered distinct from one another, as CSL Behring can benefit from the identified service either on its own or together with other resources that are readily available to CSL Behring, and as the performance obligations are separately identifiable from other performance obligations in the CSL Behring Agreement.

Refer to Note 4 “ Collaboration arrangements and concentration of credit risk ” for further detail.

Bristol-Myers Squib collaboration

The Company initially entered into collaboration, research, and license agreements with BMS in 2015 and amended them in 2020.

The Company evaluated the initial BMS CLA and determined that its performance obligations were as follows:

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Collaboration and Manufacturing Revenue

The Company recognizes Collaboration Revenues associated to optional work orders it receives from BMS to provide analytical development and process development activities that are reimbursable by BMS in accordance with the BMS CLA as well as the amended BMS CLA.

The Company recognizes Collaboration Revenues associated with optional work orders it receives from BMS to provide analytical development and process development activities that are reimbursable by BMS in accordance with the BMS CLA as well as the amended BMS CLA.

BMS and the Company entered into a Master Clinical Supply Agreement in April 2017 for the Company to supply gene therapy products during the clinical phase as well as into a binding term sheet to supply gene therapy products during the commercial phase to BMS.

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These grants generally provide for reimbursement of approved costs incurred as defined in the respective grants and are deferred and recognized in the statements of operations and comprehensive loss over the period necessary to match them with the costs they are intended to compensate, when it is probable that the Company has complied with any conditions attached to the grant and will receive the reimbursement.

The Company’s other income also consists of income from the subleasing of the Amsterdam facility while other expense consists of expenses incurred in relation to the subleasing income.

The Company’s other income also consists of employee retention credits received under the U.S.

Coronavirus Aid, Relief, and Economic Security Act, income related to a settlement agreement that the Company and VectorY B.V.

entered into in April 2021, as well as income from subleasing part of the Company’s Amsterdam facility.

Other expense consists of expenses incurred in relation to the subleasing income.

2.3.21 Research and development expenses

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2.3.23 Recently Adopted Accounting Pronouncements

Fair Value Measurement

In August 2018, the FASB issued ASU 2018-13, Fair Value Measurement (Topic 820) which modifies the disclosure requirements on fair value measurements.

ASU 2018-13 is effective for fiscal years beginning after December 15, 2019, which for the Company was January 1, 2020.

The new disclosure requirements for changes in unrealized gains and losses in other comprehensive income for recurring Level 3 measurements, the range and weighted average of significant unobservable inputs and the amended requirements for the narrative description of measurement uncertainty should be applied prospectively for only the most recent interim or annual period presented in the initial fiscal year of adoption.

All other amendments should be applied retrospectively.

ASU 2018-13 did not have a material impact on the Company’s consolidated financial statements.

Recent Accounting Pronouncements Not Yet Effective

Government Assistance

In November 2018, the FASB issued ASU 2021-10, Government Assistance (Topic 832) which discusses the requirements for disclosures related to transactions with a government.

ASU 2021-10 is effective for fiscal years beginning after December 15, 2021.

The new disclosure requirements will require disclosures around 1) information about the nature of the transactions and the related accounting policy used to account for the transactions, 2) the line items on the balance sheet and income statement that are affected by the transactions, and the amounts applicable to each financial statement line item, and 3) significant terms and conditions of the transactions, including commitments and contingencies.

An entity should apply the updates prospectively or retrospectively.

The Company currently includes information on government grants and does not expect these amendments to have a material impact on the Company’s consolidated financial statements.

Corlieve transaction

At the Acquisition Date, the Company acquired Corlieve.

Following Corlieve’s formation in November 2019, Corlieve obtained exclusive licenses to certain patents from two French research institutions that continue to collaborate with the Company.

Corlieve also obtained an exclusive license from Regenxbio Inc.

(“Regenxbio”) to use AAV9 to deliver any sequence that affects the expression of the Glutamate inotropic receptor kainate type subunit 2 (“GRIK 2”) gene sequence in humans.

Corlieve and Regenxbio simultaneously entered into a collaboration plan related to agreed joint preclinical research and development activities.

At the Acquisition Date, Corlieve and its Swiss subsidiary, Corlieve Therapeutics AG, employed seven employees.

Corlieve’s result for the full year 2021 was a $ 7.3 million loss.

The result included in the Company’s consolidated results for the year ended December 31, 2021 is a $ 4.1 million loss.

The Company evaluated the Corlieve transaction as to whether or not the transaction should be accounted for as a business combination or asset acquisition by first applying a screen test to determine if substantially all of the fair value of the gross assets acquired is concentrated in a single identifiable asset or group of similar identifiable assets.

Based on the fair values of the gross assets acquired, the Company determined the screen test was not met.

The Company further analyzed whether or not the acquired inputs and processes that have the ability to create outputs would meet the definition of a business.

Significant judgment is required in the application of the screen test to determine whether an acquisition is a business combination or an acquisition of assets.

Identifiable assets and liabilities of Corlieve, including identifiable intangible assets, were recorded at their fair values as of the Acquisition Date, when the Company obtained control.

The excess of the fair value of the consideration transferred over the fair value of the net assets acquired was recorded as goodwill.

The following table summarizes the fair values assigned to assets acquired and the liabilities assumed by the Company, along with the resulting goodwill, as of the Acquisition Date:

(in thousands)

Consideration

Contingent consideration

Liability related to Mandatorily Redeemable Shares (see below)

Fair value of total consideration

Recognized amounts of identifiable assets acquired and liabilities assumed

Current assets including € 2.8 million of cash

Property, plant and equipment

Identifiable intangible asset

Current liabilities

Deferred tax liability, net

Other non-current liabilities

Fair value of net assets acquired

Consideration

On the Acquisition Date, the Company acquired 97.7 % of the outstanding ordinary shares of Corlieve in return for EUR 44.9 million ($ 53.3 million as of the Acquisition Date).

As is contractually required the Company acquired the remaining outstanding ordinary shares on February 9, 2022 following the expiration of a minimum holding period.

The Company recorded a liability related to these Mandatorily Redeemable Shares for an amount of EUR 0.7 million ($ 0.9 million) as of the Acquisition Date.

The Company financed the Corlieve Transaction from its cash on hand.

In addition to the payments to acquire 100 % of the outstanding ordinary shares, Corlieve’s former shareholders are eligible to receive up to EUR 35.8 million ($ 40.6 million as of December 31, 2021) upon achievement of certain development milestones through Phase I/II and EUR 143.1 million ($ 162.3 million as of December 31, 2021) upon achievement of certain milestones associated with Phase III development and obtaining approval to commercialize Corlieve’s target candidate for the treatment of temporal lobe epilepsy (“AMT-260” or “TLE”) in the United States of America and the European Union.

The Company may elect to pay up to 25 % of such milestone payments through the issuance of the Company’s ordinary shares.

As of the Acquisition Date, the Company recorded EUR 20.2 million ($ 24.0 million) as a contingent liability (presented as “Non-current liability”) for the fair value of these milestone payments.

The fair value of the contingent liability as of December 31, 2021 amounted to EUR 26.0 million ($ 29.5 million).

Changes in fair value of the contingent liability are recognized within research and development expenses in the consolidated statements of operations.

Identified intangible assets

The Company determined the fair value of the IPR&D Intangible Asset using a present value model based on expected cash flows.

Estimating the amounts and timing of cash flows required to complete the development of AMT-260 as well as net sales, cost of goods sold, and sales and marketing costs involved considerable judgment and uncertainty.

The expected cash flows are materially impacted by the probability of successfully completing the various stages of development (i.e., dosing of first patient in clinical trial, advancing into late-stage clinical development and obtaining approval to commercialize a product candidate) as well as the weighted average cost of capital of 10.4 % used to discount the expected cash flows.

Based on all such information and its judgment the Company estimated the fair value of the IPR&D Intangible Asset at EUR 53.6 million ($ 63.6 million) as of the Acquisition Date.

Deferred tax liability, net

Corlieve’s deferred tax assets at the time of acquisition amounted to EUR 1.5 million ($ 1.7 million).

Recognition of the IPR&D Intangible Asset gave rise to a deferred tax liability of EUR 13.4 million ($ 15.9 million) at the enacted French corporate income tax rate of 25.0 %.

The Company consequently recorded a net deferred tax liability of EUR 11.9 million ($ 14.2 million as of the Acquisition Date).

Changes in the net deferred tax liability after the Acquisition Date will be recorded in income tax expense in the consolidated statements of operations.

Goodwill represents the excess of total consideration over the estimated fair value of net assets acquired.

The Company recorded EUR 23.9 million ($ 28.4 million) of goodwill in the consolidated balance sheet as of the Acquisition Date.

The goodwill primarily relates to the recognition of a deferred tax liability recognized in association with the IPR&D Intangible asset of EUR 13.4 million ($ 15.9 million as of Acquisition Date) as well as the fair market value of the experienced workforce and potential synergies from the acquisition.

The Company allocated the goodwill to its reporting unit.

The Company does not expect any portion of this goodwill to be deductible for income tax purposes.

As of the Acquisition Date, Corlieve held a loan with outstanding amount equal to EUR 1.0 million ($ 1.2 million), which loan was repaid in its entirety in September 2021.

As of the Acquisition Date, Corlieve also held a loan with outstanding amount equal to EUR 0.4 million ($ 0.4 million), which was repaid in its entirety in December 2021.

As of the Acquisition Date, the Company also acquired other assets and assumed other liabilities, which included among others, EUR 2.9 million ($ 3.4 million) of current assets, which consisted of EUR 2.8 million ($ 3.3 million) of cash, and EUR 1.1 million ($ 1.3 million) of current liabilities.

Collaboration arrangements and concentration of credit risk

CSL Behring collaboration

On June 24, 2020, uniQure biopharma B.V., a wholly-owned subsidiary of uniQure N.V., entered into the CSL Behring Agreement with CSL Behring LLC, (“CSL Behring”), pursuant to which CSL Behring will receive exclusive global rights to etranacogene dezaparvovec, the Company’s investigational gene therapy for patients with hemophilia B, (the “Product”).

Under the terms of the CSL Behring Agreement, the Company will receive a $ 450.0 million upfront cash payment upon the closing of the CSL Behring Agreement and be eligible to receive up to $ 1.6 billion in additional payments based on regulatory and commercial milestones.

On the Signing Date, uniQure biopharma B.V., a wholly-owned subsidiary of uniQure N.V., entered into the CSL Behring Agreement with CSL Behring, pursuant to which CSL Behring received exclusive global rights to the Product.

On May 6, 2021, a day after the expiration of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976, the CSL Behring Agreement became fully effective (“Closing”).

Pursuant to the CSL Behring Agreement, the Company received a $ 450.0 million upfront cash payment and $ 12.4 million in other payments related to the Closing and the transfer of the license.

The Company is eligible to receive up to $ 1.6 billion in additional payments based on the achievement of regulatory and commercial milestones.

The CSL Behring Agreement also provides that the Company will be eligible to receive tiered double-digit royalties in a range of up to a low-twenties percent of net sales of the Product based on sales thresholds.

Pursuant to the CSL Behring Agreement, the Company will be responsible for the completion of the HOPE-B clinical trial, manufacturing process validation, and the manufacturing supply of the Product until such time that these capabilities may be transferred to CSL Behring or its designated contract manufacturing organization.

Concurrently with the execution of the CSL Behring Agreement, the Company and CSL Behring entered into a development and commercial supply agreement, pursuant to which, among other things, the Company will supply the Product to CSL Behring at an agreed-upon price.

Clinical development and regulatory activities performed by the Company pursuant to the CSL Behring Agreement will be reimbursed by CSL Behring.

CSL Behring will be responsible for global regulatory submissions and commercialization requirements for the Product.

The effectiveness of the transactions contemplated by the CSL Behring Agreement is contingent on completion of review under antitrust laws in the United States, Australia, and the United Kingdom, and certain provisions of the CSL Behring Agreement will not become effective until after we receive all such regulatory approvals.

As of December 31, 2020, such regulatory approvals had not been received in all jurisdictions.

As of December 31, 2020, the Company concluded it has no enforceable right to the upfront payment, the regulatory and sale milestone payments, or the royalties (together “CSL Behring License Revenue”) that the Company may receive in accordance with the CSL Behring Agreement, as all payments are contingent upon the successful completion of reviews under the Hart-Scott-Rodino Antitrust Improvements Act of 1976.

Therefore, the Company determined it will not recognize any revenue in relation to the CSL Behring License Revenue, in accordance with ASC 606 during the year ended December 31, 2020.

In accordance with its existing license and other agreements, the Company is contractually required to pay in total a low to high single digit percentage of any upfront payment to its licensors and financial advisor (“License Fees”).

The Company did not record any License Fees in the year ended December 31, 2020, as the Company had not recognized the upfront payment as this date.

The Company incurred $ 2.1 million of expenses related to the obligations related to the CSL Behring Agreement that had not been satisfied as of December 31, 2020.

On the Signing Date, the Company and CSL Behring entered into a development and commercial supply agreement, pursuant to which, among other things, the Company will supply the Product to CSL Behring at an agreed-upon price commensurate with the SSP.

The Company will be responsible for supplying development and commercial Product until such time that these capabilities may be transferred to CSL Behring or a designated contract manufacturing organization.

The Company will be completing the HOPE-B clinical trial and the validation of the manufacturing process on behalf of CSL Behring, as well as provide further development services if requested by CSL Behring.

Activities related to on-demand development services as well as activities related to the completing the HOPE-B clinical trial will be reimbursed by CSL Behring at an agreed full-time-employee rate (“FTE-rate”) and CSL Behring will also reimburse agreed third-party expenses incurred in relation to performing these activities.

The validation of the manufacturing process as well as Manufacturing Development will be reimbursed through a future milestone payment.

If completed after certain contractually agreed upon dates, the milestone payment will be reduced in accordance with a pre-specified mechanism.

The Company concluded that CSL Behring is a customer in accordance with Topic 606.

The Company identified two material performance obligations related to the CSL Behring Agreement:

(i) License Sale;

(ii) Manufacturing Development.

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” and together with the License 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.

In addition, the Company concluded that variable milestone payments, sales milestone payments and royalties should be allocated to the License Sale performance obligation as well.

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 will record expenses related to its existing license and other agreements as well as its financial advisor for a high single digit percentage of any such revenue recognized associated to meeting a milestone.

The Company will include payments related to sales milestones in the transaction price when their achievement becomes probable, and it will include royalties on the sale of Product once these have been earned.

The Company determined that achievement of a total of $ 55.0 million of milestone payments related to the submissions of a biologics license application (“BLA”) and market authorization application (“MAA”) is probable as of the time of filing these financial statements and hence recorded these as license revenue in the year ended December 31, 2021.

In making the determination, the Company considered that after Closing, it believes to successfully have completed the validation of its manufacturing process for the Product in December 2021 and that CSL Behring announced the accomplishment of the primary clinical endpoint for the Product in December 2021.

The Company recognized $ 517.4 million of revenues related to the License Sale in the year ended December 31, 2021.

The Company determined that the variable milestone payment related to Manufacturing Development should be allocated to the Manufacturing Development performance obligation.

The Company concluded that this milestone payment represents the SSP of the services based on the estimated cost of providing the services including a reasonable margin.

The services related to Manufacturing Development will be provided between Closing and the completion of an agreed manufacturing development plan.

The variable consideration will be reduced if the Company does not complete the development by pre-agreed dates.

The Company utilized the most likely amount method to estimate the variable consideration to be included in the transaction price.

Completion of Manufacturing Development is partially dependent on the timing of regulatory submissions by CSL Behring as well as regulatory approvals of the developed manufacturing processes.

Since the Company cannot control the timing or outcome of any regulatory decisions, the Company concluded that it would recognize revenue related to this payment when it becomes probable that the milestone has been achieved.

The Company has not recognized any revenue related to Manufacturing Development.

The Company recognized $ 2.4 million of collaboration revenue in the year ended December 31, 2021, respectively, compared to nil in the same periods in 2020 and 2019.

The Company generates such collaboration revenue from services rendered in relation to completing the HOPE-B clinical trial on behalf of CSL Behring.

CSL Behring may request additional development services or request the Company to support the transfer of manufacturing to a party designated by CSL Behring.

These collaboration services will be reimbursed at the pre-agreed FTE-rate.

The Company concluded that these rights at Closing do not represent material rights.

The Company incurred $ 2.1 million of expenses for obligations related to the CSL Behring Agreement that had not been satisfied as of December 31, 2020.

The Company capitalized these expenses as contract fulfillment costs (presented within Other current assets).

As of December 31, 2020, the Company also recognized a $ 2.1 million receivable (presented within Accounts receivable) from CSL Behring for expenses for which it has a right of reimbursement as well as a contract liability (presented within Accrued expenses and other current liabilities) for the same amount.

In accordance with ASC 606 it cannot recognize any CSL Behring License Revenue as of this date.

In accordance with ASC 606 the Company could not recognize any license revenue related to the CSL Behring Agreement in the period ended December 31, 2020.

Following the Closing, the Company collected the $ 2.1 million of accounts receivable related to reimbursable contract fulfillment costs that was outstanding as of December 31, 2020 .

As of December 31, 2021, the Company has recorded accounts receivable of $ 2.9 million from CSL Behring related to clinical development services as well as a contract asset of $ 55.0 million related to BLA and MAA submission milestone payments considered probable.

Bristol-Myers Squibb collaboration

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The BMS CLA initially provided that the Company and BMS could potentially have collaborated on up to ten Collaboration Targets in total.

BMS initially designated four Collaboration Targets, including S100A1 for congestive heart failure (“AMT-126”).

In October 2018, the Company and BMS completed a heart function proof-of-concept study of AMT-126 in a pre-clinical, diseased animal model.

The data did not show a benefit on heart function at six months and, consequently, work on S100A1 was discontinued.

The Company impaired a $ 5.4 million acquired research and development asset associated with the program and released a contingent liability of $ 3.8 million related to the acquisition of the asset to income in the year ended December 31, 2018.

In April 2019, BMS designated a new cardiovascular Collaboration Target to replace S100A1.

As a result, BMS had designated a total of four Collaboration Targets as of December 31, 2019.

2020 Amendment

In February 2019, BMS requested a one-year extension of the initial research term.

In April 2019, following an assessment of the progress of the collaboration and the Company’s expanding proprietary programs, the Company notified BMS that the Company did not intend to agree to an extension of the initial research term but rather preferred to restructure or amend the collaboration to reduce or eliminate certain of the Company’s obligations under it.

On December 1, 2020, the Company and BMS entered into the amended BMS CLA.

Under the amended BMS CLA, BMS is limited to four Collaboration Targets.

For a period of one-year from the effective date of the amended BMS CLA, BMS may replace up to two of these four Collaboration Targets with up to two new targets in the field of cardiovascular disease.

For a period of one-year from the effective date of the amended BMS CLA, BMS was able to replace up to two of the four active Collaboration Targets with two new targets in the field of cardiovascular disease.

The Company continues to be eligible to receive research, development, and regulatory milestone payments of up to $ 217.0 million for each Collaboration Target, if defined milestones are achieved.

BMS is no longer entitled to designate the fifth to tenth Collaboration Targets and as such the Company’s remaining obligations under the amended BMS CLA are substantially reduced.

The Company is no longer entitled to receive up to an aggregate $ 16.5 million in target designation payments for the research, development and regulatory milestone payments associated with the fifth to tenth Collaboration Targets.

Since the December 2020 amendment, BMS is no longer entitled to designate the fifth to tenth Collaboration Targets and as such the Company’s remaining obligations under the amended BMS CLA are substantially reduced.

The Company is also no longer entitled to receive up to an aggregate $ 16.5 million in target designation payments for the research, development and regulatory milestone payments associated with the fifth to tenth Collaboration Targets.

For as long as any of the four Collaboration Targets are being advanced, BMS may place a purchase order to be supplied with research, clinical and commercial supplies.

1 unchanged sentence

Both BMS and the Company may agree to a technology transfer of manufacturing capabilities pursuant to the terms of the amended BMS CLA.

The amended BMS CLA does not extend the initial research term.

The amended BMS CLA does not extend the initial four-year research term that expired in May 2019.

BMS may place purchase orders to provide limited services primarily related to analytical and development efforts in respect of the four Collaboration Targets.

BMS may request such services for a period not to exceed the earlier of (i) the completion of all activities under a Research Plan and (ii) either (A) three years after the last replacement target has been designated by BMS during the one year replacement period following the amended BMS CLA effective date or (B) three years if no replacement targets are designated during this one year period and BMS continues to reimburse the Company for these services.

The Company evaluated the impact of the amendment of the BMS CLA in relation to its performance obligation related to:

– Providing access to its technology and know-how in the field of gene therapy and participating in joint steering committee and other governing bodies (materially satisfied as of December 1, 2020) (“License Revenue”).

BMS may request such services for a period not to exceed the earlier of (i) the completion of all activities under a Research Plan and (ii) November 30, 2023, if no replacement targets are designated.

BMS continues to reimburse the Company for these services.

During the year ended December 31, 2020, the Company evaluated the impact of the amendment of the BMS CLA had in relation to its performance obligation related to License Revenue.

The Company did not identify any new distinct performance obligations and determined the amended BMS CLA did not represent a separate contract in accordance with ASC 606.

The Company evaluated the effect the modification has on its measure of progress towards the completion of its performance obligation related to License Revenue and recorded an adjustment to License Revenue as of December 1, 2020.

The Company evaluated the effect the modification had on its measure of progress towards the completion of its performance obligation related to License Revenue and determined that its remaining performance obligation under the amended BMS CLA was immaterial and recognized the remaining balance of unrecognized License Revenue as of November 30, 2020.

Services to BMS are rendered by the Dutch operating entity.

Total collaboration and license revenue generated with BMS are as follows (presented as revenue from a related party until November 30, 2020, and as revenue from December 1, 2020 onwards):

Total collaboration and license revenue generated with BMS are as follows (presented as revenue from a related party up until the effective date of the amended BMS CLA and presented as revenue after the effective date):

Years ended December 31,

1 unchanged sentence

Bristol Myers Squibb

Amounts owed by BMS in relation to the Collaboration and License Revenue are as follows (presented as “Accounts receivables” as of December 31, 2020 and as “Accounts receivable from related party” as of December 31, 2019):

Amounts owed by BMS in relation to the Collaboration and License Revenue are as follows (presented as “Accounts receivables” as of December 31, 2021 and 2020:

(in thousands)

8 unchanged sentences

License Revenue

The Company recognized $ 33.0 million of License Revenue for the year ended December 31, 2020 (December 31, 2019:

The Company recognized no License Revenue for the year ended December 31, 2021 (December 31, 2020:

$ 33.0 million, December 31, 2019:

1 unchanged sentence

On May 21, 2015, the Company recorded a $ 60.1 million upfront payment and in August 2015 it recorded a $ 15.0 million payment it received from BMS in relation to the designation of the second, third and fourth Collaboration Targets.

The Company recognizes License Revenue over the expected performance period based on its measure of progress towards the completion of certain activities related to its services.

The Company determines such progress by comparing activities performed at the end of each reporting period with total activities expected to be performed.

The Company estimates total expected activities using several unobservable inputs, such as the probability of BMS designating additional targets, the probability of successfully completing each phase and estimated time required to provide services during the various development stages.

The estimation of total services at the end of each reporting period involves considerable judgement.

The Company recognized License Revenue over the expected performance period based on its measure of progress towards the completion of certain activities related to its services.

The Company determined such progress by comparing activities performed at the end of each reporting period with total activities expected to be performed.

The Company estimated total expected activities using several unobservable inputs, such as the probability of BMS designating additional targets, the probability of successfully completing each phase and estimated time required to provide services during the various development stages.

The estimation of total services at the end of each reporting period involves considerable judgment.

The amount of services the Company expects to provide is significantly impacted by the number of Collaboration Targets that it estimates BMS would pursue.

4 unchanged sentences

The Company includes variable consideration related to any research, development, and regulatory milestone payments, in the transaction price once it is considered probable that including these payments in the transaction price would not result in the reversal of cumulative revenue recognized.

Due to the significant uncertainty surrounding the development of gene-therapy product candidates and the dependence on BMS’s performance and decisions, the Company does not currently (with below exception) consider this probable.

On December 17, 2020 BMS designated one of the four Collaboration Targets as a candidate to advance into IND-enabling studies entitling the Company to receive a $ 4.4 million research milestone payment.

The Company recorded the $ 4.4 million as License Revenue in the twelve-month period ended December 31, 2020.

However, there was a milestone that was recorded as license revenue in the year ended December 31, 2020 (see below).

On December 17, 2020 BMS designated one of the four Collaboration Targets as a candidate to advance into Investigational New Drug-enabling studies (“IND-enabling studies”) entitling the Company to receive a $ 4.4 million research milestone payment.

The Company recorded the $ 4.4 million as License Revenue in the year ended December 31, 2020.

The Company recognizes License Revenue related to product sales by BMS from any of the Collaboration Targets when the sales occur.

1 unchanged sentence

The royalty term is determined on a licensed-product-by-licensed-product and country-by-country basis and begins on the first commercial sale of a licensed product in a country and ends on the expiration of the last to expire of specified patents or regulatory exclusivity covering such licensed product in such country or, with a customary royalty reduction, ten years after the first commercial sale if there is no such exclusivity.

Fair value measurement

Fair value measurement and Other non-operating (losses) / gains

The Company measures certain financial assets and liabilities at fair value, either upon initial recognition or for subsequent accounting or reporting.

The Company’s only material financial assets measured at fair value using Level 1 inputs is cash and cash equivalents.

The liability measured at fair value using Level 3 inputs as of December 31, 2020 was the derivative financial instrument.

The Company had recorded derivative financial instruments as of December 31, 2019, that were measured at fair value using Level 3 inputs.

Changes in Level 3 items during the years ended December 31, 2020, 2019 and 2018 are as follows:

The Company’s only material financial assets measured at fair value using Level 1 inputs is cash and cash equivalents and restricted cash.

Restricted cash is included within “Other non-current assets” within the consolidated balance sheets.

The following table sets forth the balances and changes in fair values of liabilities that are measured at fair value using Level 3 inputs:

consideration

1 unchanged sentence

Balance at December 31, 2018

Net gains recognized in profit or loss

Currency translation effects

Balance at December 31, 2018

Net losses recognized in profit or loss

3 unchanged sentences

Net gains recognized in profit or loss

Derecognition of warrants

Derecognition of BMS warrants

Recognition of derivative financial liability of CoC-payment

1 unchanged sentence

Balance at December 31, 2020

Amount recorded for contingent consideration on Acquisition Date of Corlieve

Net losses recognized in profit or loss

Currency translation effects

Balance at December 31, 2021

Derivative financial instruments

The Company issued derivative financial instruments related to its collaboration with BMS and in relation to the issuance of the Hercules loan facility.

The Company recorded the following results in other non-operating (losses) / gains related to the changes in the fair value of derivative financial instruments.

Years ended December 31,

(in thousands)

Other non-operating gains:

Derivative gains

Total other non-operating gains:

Other non-operating losses:

Derivative losses

Total other non-operating losses:

Other non-operating (losses) / gains, net

Derivative financial instruments BMS

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The fair value of the warrants as of December 31, 2019 was $ 3.1 million.

During the year ended December 31, 2020, the Company recognized a $ 3.1 million gain in non-operating income / expense (December 31, 2019:

$ 2.3 million loss;

December 31, 2018:

$ 0.5 million gain) related to fair value changes of the BMS warrants.

During the year ended December 31, 2020, the Company recognized a $ 3.1 million gain in non-operating (losses) / gains (December 31, 2019:

$ 2.3 million loss) related to fair value changes of the BMS warrants.

The gain recognized in the year ended December 31, 2020 includes $ 0.8 million from the derecognition of the BMS warrants on December 1, 2020.

5 unchanged sentences

The probability of the occurrence of these events represented another significant unobservable input used in the calculation of the fair value of the warrants.

On December 1, 2020, the Company and BMS terminated the BMS warrants and 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, uniQure (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”).

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 December 31, 2021 that would obligate it to make a CoC-payment.

3 unchanged sentences

The Company determined 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 event within the biotechnology industry.

The Company estimated this unobservable input using the best information available as of December 1, 2020 and December 31, 2020.

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 December 1, 2020, and December 31, 2020 and 2021, respectively.

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 December 1, 2020 and December 31, 2020 was $ 2.6 million.

The Company recorded a $ 2.6 million loss within “Other non-operating expenses” in the twelve-month period ended December 31, 2020 related to the initial recognition of this derivative financial liability.

Selecting and evaluating market information involves considerable judgment 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 December 31, 2021 was $ 2.8 million (December 1, 2020 and December 31, 2020:

$ 2.6 million).

The Company recorded a $ 2.6 million loss within “Other non-operating (losses) / gains” in the year ended December 31, 2020 related to the initial recognition of this derivative financial liability.

The increase of the fair market value of the derivative financial liability of $ 0.2 million in the year ended December 31, 2021 was recorded in Other non-operating (losses) / gains.

Hercules loan facility

On June 14, 2013, the Company entered into a venture debt loan facility (the “Original Facility”) with Hercules (see Note 8, “Long-term debt”) pursuant to a Loan and Security Agreement (the “Loan Agreement”), which included a warrant maturing on February 5, 2019.

In 2013 the Company entered into a venture debt loan facility with Hercules (see Note 10, “ Long-term debt ”) which included a warrant maturing on February 5, 2019.

The warrant was not closely related to the host contract and was accounted for separately as a derivative financial liability measured at fair value though profit or loss.

The warrant included in the Original Facility remained in place following the 2014, 2016 and 2018 amendments of the loan.

The Hercules warrants were exercised as of February 1, 2019.

The Company issued 37,175 ordinary shares at $ 34.25 following the exercise of all Hercules warrants and receipt of $ 0.5 million from Hercules.

During the year ended December 31, 2020, the Company recognized no more gains or losses in other non-operating income / (expense) (December 31, 2019:

During the years ended December 31, 2021 and 2020, respectively, the Company recognized no more gains or losses in Other non-operating (losses) / gains related to fair value changes of the Hercules warrants (December 31, 2019:

$ 0.2 million loss).

December 31, 2018:

$ 0.3 million loss) related to fair value changes of the Hercules warrants.

Contingent consideration

In connection with the Company’s acquisition of the InoCard business (“InoCard”) in 2014, the Company recorded contingent consideration related to amounts potentially payable to InoCard’s former shareholders.

The amounts payable in accordance with the sale and purchase agreement (as amended in August 2017) were contingent upon realization of milestones associated with its S100A1 protein research program.

Following the discontinuation of the research program the Company since 2018 no longer expects to realize those milestones and recorded a $ 3.8 million gain within research and development expenses for the year ended December 31, 2018, to release the liability to profit and loss.

Corlieve transaction

The Company is required to pay up to EUR 178.8 million ($ 202.8 million at the December 31, 2021 foreign exchange rate) to the former shareholders of Corlieve upon the achievement of contractually defined milestones in connection with the Company’s acquisition of Corlieve (refer to Note 3 “Corlieve transaction” ).

The Company recorded a liability for the fair market value of the contingent consideration of EUR 20.2 million ($ 24.0 million) at the Acquisition Date.

The fair market value 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.

This discount rate was determined using the effective interest rate of the Company’s existing debt facility adjusted for difference in maturity dates based on CCC yield curve.

The fair value of the contingent consideration as of December 31, 2021 was $ 29.5 million using discount rates ranging from 10.9 % to 11.9 % as well as a 55 % likelihood of AMT-260 advancing into clinical development by no later than early 2024.

The Company increased the likelihood of advancing into clinical development from 40 % to 55 % following the designation of a lead candidate in late October 2021, which resulted in EUR 5.0 million ($ 5.8 million) increase of the contingent liability.

If as of December 31, 2021 the Company had assumed a 100 % likelihood of AMT-260 advancing into clinical development, then the fair value of the contingent consideration would have increased to $ 47.0 million.

If as of December 31, 2021 the Company 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 liability are recognized within research and development expenses in the consolidated statements of operations.

As of December 31, 2021, the Company recorded $ 0.8 million related to consideration for post-acquisition services, presented within Other non-current liabilities in connection with the Company’s acquisition of Corlieve.

Property, plant, and equipment, net

18 unchanged sentences

Right-of-use asset and lease liabilities

The Company adopted ASU 2016-02 “Leases (Topic 842)” as well as ASU 2018-10 and ASU 2018-11, which both relate to improvements to ASC 842.

The Company adopted the standard using the modified retrospective approach with an effective date as of the beginning of the Company’s fiscal year, January 1, 2019 (“new lease accounting standard”).

The standard requires the balance sheet recognition for leases.

Prior years were not recast under the new standard and therefore, those amounts are presented in accordance with the requirements of the previously effective lease standard ASC 840 (“historic lease accounting standard”).

The Company elected to utilize practical expedients available for expired or existing contracts which allowed the Company to carryforward historical assessments of (1) whether contracts are or contain leases, (2) lease classification, and (3) initial direct costs.

The Company’s most significant leases relate to office and laboratory space under the following operating lease agreements:

8 unchanged sentences

The contractually fixed annual increase of lease payments through 2029 for both the extension and expansion lease have been included in the lease payments.

In December 2021, the Company entered into a new lease for an additional facility in Lexington, Massachusetts, United States of approximately 13,501 square feet of space.

The lease is expected to commence in the second half of 2022, is set for seven years starting from the rent commencement date and is non-cancellable.

The lease is renewable for one five-year term.

Amsterdam / The Netherlands

In March 2016, the Company entered into a 16-year lease for a facility in Amsterdam, the Netherlands and amended this agreement in June 2016.

The Company consolidated its three Amsterdam sites into the new site at the end of May 2017.

The lease for the new facility terminates in 2032, with an option to extend in increments of five-year periods.

The lease for the facility terminates in 2032, with an option to extend in increments of five-year periods.

The lease contract includes variable lease payments related to annual increases in payments based on a consumer price index.

2 unchanged sentences

The fixed lease payments to be received during the remaining term under the agreement to sub-lease amount to $ 5.4 million (EUR 4.7 million) as of December 31, 2021.

In May 2021, the Company entered into a sublease agreement to let an additional approximately 1,080 square meters of office space to accommodate the hiring of additional full-time employees.

The lease expires in October 2028 and includes an option to break the lease on October 31, 2023.

Operating lease liabilities

6 unchanged sentences

Total lease cost

The rent expense in accordance with the historical lease accounting standard for the year ended December 31, 2018, was calculated on a straight-line basis over the term of the lease and considered $ 12.2 million of lease incentives received.

Aggregate rent expense was as follows:

Year ended December 31,

(in thousands)

Rent expense - Lexington

Rent expense - Amsterdam

Total lease cost

The table below presents the lease-related assets and liabilities recorded on the Consolidate balance sheets in accordance with the new lease accounting standard.

The table below presents the lease-related assets and liabilities recorded on the Consolidated balance sheets in accordance with the new lease accounting standard.

(in thousands)

5 unchanged sentences

The weighted-average remaining lease term as of December 31, 2021, is 8.3 years, compared to 9.4 years as of December 31, 2020, and the weighted-average discount rate as of December 31, 2021 is 11.34 % , compared to 11.37 % as of December 31, 2020.

The Company derived the weighted-average discount rate, adjusted for differences such as in the term and payment patterns, from the Company’s loan from Hercules capital which was refinanced immediately prior to the January 1, 2019, adoption date in December 2018.

The table below presents supplemental cash flow and non-cash information related to leases required in accordance with the new lease accounting standard.

The Company uses an incremental borrowing rate applicable to the lease asset.

The table below presents supplemental cash flow and non-cash information related to leases.

Year ended December 31,

(in thousands)

Cash paid for amounts included in the measurement of lease liabilities

Operating cash flows for operating leases 1)

(1) The Company has received $ 1.5 million of landlord incentive payments for the year ended December 31, 2019, which are not included in the cash paid amounts.)

The Company did not obtain any right-of-use assets in exchange for the lease obligations during the year ended December 31, 2020.

Besides the initial recognition of operating right-of-use assets of $ 19.0 million upon adoption of the new lease standards on January 1, 2019, the Company obtained $ 9.0 million of additional right-of-use assets in exchange for lease obligations during the year ended December 31, 2019.

Right-of-use asset obtained in exchange for lease obligation

Operating lease 2)

1) The Company received $ 1.5 million of landlord incentive payments for the year ended December 31, 2019, which are not included in the cash paid amounts.

2) The Company capitalized $ 19.0 million of operating right-of-use assets upon adoption of ASC 842 Leases on January 1, 2019 that are not included in the movement for the year ended December 31, 2019.

Undiscounted cash flows

The table below reconciles the undiscounted cash flows as of December 31, 2020, for each of the first five years and the total of the remaining years to the operating lease liabilities recorded on the Consolidated balance sheet as of December 31, 2020 in accordance with the new lease accounting standard.

The table below reconciles the undiscounted cash flows as of December 31, 2021, for each of the first five years and the total of the remaining years to the operating lease liabilities recorded on the Consolidated balance sheet as of December 31, 2021.

Amsterdam (1)

6 unchanged sentences

(1) Payments are due in EUR and have been translated at the foreign exchange rate as of December 31, 2021, of $ 1.13 / €1.00)

Intangible assets

Acquired licenses

The following table presents the Company’s acquired licenses as of December 31:

Intangible assets, net and Goodwill

The following table presents the Company’s acquired licenses and acquired IPR&D as of December 31:

(in thousands)

Less accumulated amortization and impairment

Licenses, net

All intangible assets are owned by uniQure biopharma B.V, a subsidiary of the Company.

Acquired licenses

Less accumulated amortization

Acquired licenses, net

Acquired IPR&D Intangible Asset

Intangibles, net

Acquired licenses

All acquired licenses are owned by uniQure biopharma B.V, a subsidiary of the Company.

The acquired licenses have a weighted average remaining life of 10.8 years as of December 31, 2021.

1 unchanged sentence

During the same period, the Company disposed of a number of licenses determined to have no alternative future use.

During the year ended December 31, 2019, the Company capitalized $ 1.0 million of expenditures related to contractual milestone payments under existing license agreements.

As of December 31, 2021, the estimated future amortization expense for each of the five succeeding years and the period thereafter is as follows:

7 unchanged sentences

December 31, 2019 $ 0.0 million).

Acquired research and development

The Company acquired research and development assets as part of its acquisition of InoCard in July 2014.

Based on the review of pre-clinical data associated with those assets, the Company does not expect that it will pursue further research related to those assets.

Accordingly, the Company recorded a $ 5.4 million impairment loss within research and development expenses in the year ended December 31, 2018, to reduce the asset’s carrying amount to its fair value of nil .

Acquired in-process research and development

As part of its acquisition of Corlieve as of July 30, 2021, the Company identified certain intangible assets related to an IPR&D Intangible Asset.

Refer to Note 3 “Corlieve transaction” .

As part of its acquisition of Corlieve as of July 30, 2021, the Company recorded goodwill.

Refer to Note 3 “Corlieve transaction” .

Accrued expenses and other current liabilities

1 unchanged sentence

(in thousands)

Accruals for services provided by vendors-not yet billed

Accruals for goods received from and services provided by vendors-not yet billed

Personnel related accruals and liabilities

Accrued contract fulfillment costs and costs to obtain a contract

Contract liability (see Note 4.

Collaboration arrangements)

"Collaboration arrangements and concentration of credit risk")

Long-term debt

On June 14, 2013, the Company entered into a venture debt loan facility with Hercules, which was amended and restated on June 26, 2014, and again on May 6, 2016 (“2016 Amended Facility”).

On December 6, 2018, the Company signed an amendment to the Second Amended and Restated Loan and Security Agreement that both refinanced the then-existing $ 20.0 million 2016 Amended Facility and provided an additional unconditional commitment of $ 15.0 million as well as a conditional commitment of $ 15.0 million that expired on June 30, 2020 (the “2018 Amended Facility”).

At signing of the 2018 Amended Facility, the Company drew down an additional $ 15.0 million for a total of $ 35.0 million outstanding.

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”), which was amended and restated on June 26, 2014, and again on May 6, 2016 (“2016 Amended Facility”).

On December 6, 2018, the Company signed an amendment that both refinanced the then-existing $ 20.0 million 2016 Amended Facility and allowed the Company to draw down an additional $ 15.0 million (“2018 Amended Facility”).

The 2018 Amended Facility extended the loan’s maturity date from May 1, 2020 until June 1, 2023.

The interest-only period was initially extended from November 2018 to January 1, 2021, and was further extended to January 1, 2022, as a result of meeting the provision in the 2018 Amended Facility of raising more than $ 90.0 million in equity financing in September 2019.

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 interest rate continues to be adjustable and is the greater of (i) 8.85 % or (ii) 8.85 % plus the prime rate less 5.50 % per annum.

Under the 2018 Amended Facility, the Company paid a facility fee of 0.50 % of the $ 35.0 million outstanding as of signing and owes a back-end fee of 4.95 % of the outstanding debt.

The interest rate is adjustable and is the greater of (i) 8.85 % and (ii) 8.85 % plus the prime rate less 5.50 % per annum.

Under the 2018 Amended Facility, the Company owes a back-end fee of 4.95 % of the outstanding debt.

In addition, in May 2020 the Company paid a back-end fee of $ 1.0 million in relation to the 2016 Amended Facility.

The amortized cost (including interest due presented as part of accrued expenses and other current liabilities) of the 2018 Amended Facility was $ 35.9 million as of December 31, 2020, compared to $ 36.3 million as of December 31, 2019, and is recorded net of discount and debt issuance costs.

The foreign currency gain on the loan was $ 3.1 million in 2020 (2019:

loss of $ 0.7 million;

On January 29, 2021, the Company and Hercules amended the 2018 Amended Facility (“2021 Amended Facility”).

Pursuant to the 2021 Amended Facility, Hercules agreed to an additional Facility of $ 100.0 million (“Tranche B”) increasing the aggregate principal amount of the term loan facilities from $ 35.0 million to up to $ 135.0 million.

On January 29, 2021, the Company drew down $ 35.0 million of the Tranche B.

Advances under Tranche B bore interest at a rate equal to the greater of (i) 8.25 % or (ii) 8.25 % plus the prime rate, less 3.25 % per annum.

The principal balance of $ 70.0 million and all accrued but unpaid interest on advances under Tranche B was due on June 1, 2023, which date could had been extended by the Company by up to two twelve-month periods.

Advances under the 2021 Amended Facility could have been prepaid without charge after July 29, 2021.

The back-end fee in respect of advances under the 2021 Amended Facility ranged from 1.65 % to 6.85 %, depending on the repayment date.

In addition to Tranche B, the 2021 Amended Facility had also extended the interest only payment period of the previously funded $ 35.0 million term loan (“Tranche A”) from January 1, 2022 to June l, 2023.

On December 15, 2021, the Company and Hercules amended and restated the 2021 Amended Facility (“2021 Restated Facility”).

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, resulting in total principal outstanding as of December 31, 2021 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 is extended from January 1, 2023 to December 1, 2024, or December 1, 2025 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 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 Amended Facility was $ 101.6 million as of December 31, 2021, compared to an amortized cost of $ 35.9 million for the 2018 Amended Facility as of December 31, 2020, and is recorded net of discount and debt issuance costs.

The foreign currency loss on the loan was $ 5.3 million in 2021 (2020:

gain of $ 3.1 million;

loss of $ 0.7 million).

3 unchanged sentences

(in millions)

As a covenant in the 2018 Amended 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.

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.

In combination with other covenants, the 2018 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.

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 $ 809.2 million with the exception of $ 103.2 million of cash and cash equivalents and other current assets held by uniQure N.V.

The 2018 Amended 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 December 31, 2020, the Company was in compliance with all covenants and provisions.

The aggregate maturities of the loan, including $ 7.4 million of coupon interest payments and financing fees, for each of the 29 months after December 31, 2020, are as follows (prior to the January 2021 loan amendment – see Note 18, “Subsequent events”):

The 2021 Restated Facility contain 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 December 31, 2021, the Company was in material compliance with all covenants and provisions.

The aggregate maturities of the loans, including $ 35.6 million of coupon interest payments and financing fees, for each of the 47 months after December 31, 2021, are as follows:

(in thousands)

1 unchanged sentence

As of December 31, 2021, the Company’s authorized share capital is € 4.0 million (or $ 4.5 million when translated at an exchange rate as of December 31, 2021, of $ 1.13 / €1.00), divided into 80,000,000 ordinary shares, each with a nominal value of € 0.05 .

Under Dutch law, the authorized share capital is the maximum capital that the Company may issue without amending its articles of association.

The Company’s shareholders, at the 2021 Annual General Meeting of Stockholders held on June 16, 2021, approved an increase in the number of authorized ordinary shares by 20,000,000 to 80,000,000 million.

All ordinary shares issued by the Company were fully paid.

Besides the minimum amount of share capital to be held under Dutch law, there are no distribution restrictions applicable to the equity of the Company.

As of December 31, 2020, and 2019 and 2018 the Company’s reserves were restricted for payment of dividends for an accumulated foreign currency translation gain of $ 9.9 million in 2020 and accumulated foreign currency translation losses of $ 6.7 million and $ 7.3 million in 2019 and 2018, respectively.

As of December 31, 2021, and 2020 and 2019 the Company’s other comprehensive result was restricted for payment of dividends for an accumulated other comprehensive loss of $ 28.9 million in 2021, an accumulated other comprehensive gain of $ 9.9 million in 2020, and an accumulated other comprehensive loss of $ 6.7 million in 2019.

On March 1, 2021, the Company entered into a Sales Agreement with SVB Leerink LLC (“SVB Leerink”) with respect to an at-the-market (“ATM”) offering program, under which the Company may, from time to time in its sole discretion, offer and sell through SVB Leerink, acting as agent, its ordinary shares, up to an aggregate offering price of $ 200.0 million.

The Company will pay SVB Leerink a commission equal to 3 % of the gross proceeds of the sales price of all ordinary shares sold through it as sales agent under the Sales Agreement.

In March and April 2021, the Company issued an aggregate of 921,730 ordinary shares at a weighted average price of $ 33.52 per ordinary share, with net proceeds of $ 29.6 million, after deducting underwriting discounts and net of offering expenses.

The Company defers direct, incremental costs associated to this offering, except for the commission costs to SVB Leerink, which are a reduction to additional paid-in capital, and will deduct these costs from additional paid-in capital in the consolidated balance sheets proportionately to the amount of proceeds raised.

During the year ended December 31, 2021, $ 1.3 million of direct, incremental costs were deducted from additional paid-in capital.

Following the Closing of the CSL Behring transaction, the Company consumed its tax net operating loss carryforwards from the years 2011 to 2018.

The Company allocated the tax benefit from the release of the valuation allowance related to net operating loss carryforwards generated by share issuance costs incurred in 2014, 2015, 2017 and 2018 to additional paid-in capital.

This resulted in an increase of additional paid-in capital of $ 3.0 million in the year ended December 31, 2021.

On September 10, 2019, the Company completed a follow-on public offering of 4,891,305 ordinary shares at a public offering price of $ 46.00 per ordinary share, and on September 13, 2019, the Company completed the sale of an additional 733,695 ordinary shares at a public offering price of $ 46.00 per ordinary share pursuant to the exercise by the underwriters of the option to purchase additional ordinary shares, resulting in total gross proceeds to the Company of $ 258.8 million.

1 unchanged sentence

The Company deducted $ 0.6 million of expenses incurred related to this offering from additional paid-in capital in the accompanying consolidated balance sheets and reflected this within the proceeds from public offering of shares, net of issuance costs within the cash flows from financing activities.

On May 7, 2018, the Company completed a follow-on public offering of 5,175,000 ordinary shares at a public offering price of $ 28.50 per ordinary share, resulting in gross proceeds to the Company of $ 147.5 million.

The net proceeds to the Company from this offering were $ 138.4 million, after deducting underwriting discounts and commissions and other offering expenses payable by the Company.

The Company deducted $ 0.2 million of expenses incurred related to this offering from additional paid-in capital in the accompanying consolidated balance sheet and reflected this within the proceeds from public offering of shares, net of issuance costs within the cash flows from financing activities.

In February 2019, the Company issued 37,175 ordinary shares to Hercules pursuant to exercised warrants for $ 0.5 million in aggregate cash consideration.

25 unchanged sentences

At the general meeting of shareholders on January 9, 2014, the Company’s shareholders approved the adoption of the 2014 Plan.

At the annual general meetings of shareholders in June 2015, 2016 and 2018, uniQure shareholders approved amendments of the 2014 Plan, increasing the shares authorized for issuance by 1,070,000 shares in 2015, 3,000,000 in 2016 and 3,000,000 shares in 2018, for a total of 8,601,471 shares.

At the annual general meetings of shareholders in June 2015, 2016, 2018 and 2021, uniQure shareholders approved amendments of the 2014 Plan, increasing the shares authorized for issuance by 1,070,000 shares in 2015, 3,000,000 in 2016, 3,000,000 shares in 2018 and 4,000,000 shares in 2021 for a total of 12,601,471 shares.

Share options

72 unchanged sentences

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

In January 2019, the Company awarded PSUs to its executives and other members of senior management.

These PSUs were earned in January 2020 based on the Board’s assessment of the level of achievement of agreed upon performance targets through December 31, 2019.

The PSUs awarded for the year ended December 31, 2019 will vest on the third anniversary of the grant, subject to the grantee’s continued employment.

The following table summarizes information about the weighted average grant-date fair value, determined at of the date these were earned, of PSUs granted during the years ended December 31:

The Company granted shares to certain employees in September and December 2021 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 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.

In January 2018 and January and February 2019, the Company awarded PSUs to its executives and other members of senior management.

These PSUs were earned in January 2019 and January 2020, based on the Board’s assessment of the level of achievement of agreed upon performance targets through December 31, 2018, and December 31, 2019, respectively.

The PSUs awarded for the year ended December 31, 2018 vested in February 2021 and the PSUs awarded for the year ended December 31, 2019 vested in January 2022.

The following table summarizes information about the weighted average grant-date fair value of the PSUs determined as of the date these were earned for the 2018 and 2019 PSUs, and the date of the grant for the 2021 PSUs:

Weighted average

18 unchanged sentences

Laboratory and development expenses

Office and housing expenses

Legal and advisory expenses

Depreciation, amortization, and impairment expenses

Patent and license expenses

Office and housing expenses

Other operating expenses

Depreciation and amortization expenses

Fair value loss - Corlieve contingent consideration

Patent and license expenses

Details of employee-related expenses for the years ended December 31 are as follows:

3 unchanged sentences

Share-based compensation expenses

Consultant expenses

Other employee expenses

Social security costs

Contractor expenses

Health insurance

Pension costs - defined contribution plans

Other employee expenses

Other non-operating income / (expense)

Other non-operating income / (expense) consists of changes in the fair value of derivative financial instruments (see Note 4, “Fair value measurement”).

Years ended December 31,

(in thousands)

Other non-operating income:

Derivative gains

Total other non-operating income:

Other non-operating expense:

Derivative losses

Total other non-operating expense:

Other non-operating income / (expense), net

The Company recorded a net gain of $ 0.5 million for the year ended December 31, 2020, compared to a net loss of $ 2.3 million and a net gain of $ 0.5 million for the years ended December 31, 2019 and December 31, 2018, respectively, related to the derivative financial instruments issued as part of its collaboration with BMS.

The $ 0.5 million gain for the year ended December 31, 2020 includes a $ 2.3 million gain related to the reduction of the fair market value of the BMS warrants, a $ 0.8 million gain related to the derecognition of the BMS warrants on December 1, 2020 and a $ 2.6 million loss related to the initial recognition of the derivative financial liability related to the CoC-payment.

The Company recorded a net loss of $ 0.2 million and $ 0.3 million for the years ended December 31, 2019 and December 31, 2018, respectively, related to warrants issued to Hercules.

Income tax benefit / (expense)

No current tax expense or liabilities were recorded in 2020 by the Company’s Dutch and U.S entities.

Due to the uncertainty surrounding the realization of favorable tax attributes in future tax returns, the Company has recorded a full valuation allowance against the Company’s net deferred tax assets in the Netherlands.

The Company released the full valuation allowance against the Company’s net deferred tax assets in the United States as of December 31, 2020.

Other income during the year ended December 31, 2021 was $ 12.3 million compared to $ 3.3 million and $ 1.9 million during the same periods in 2020 and 2019, respectively.

Other income in 2021, 2020 and 2019 includes income from payments received from European authorities to subsidize the Company’s research and development efforts in the Netherlands.

The amount recognized in the year ended December 31, 2021 was $ 5.3 million compared to $ 1.9 million in 2020 and $ 0.7 million in 2019.

In addition, other income includes $ 2.6 million of employee retention credits received under the U.S.

Coronavirus Aid, Relief, and Economic Security Act, during the year ended December 31, 2021.

An additional $ 3.0 million of other income was recorded in the year ended December 31, 2021, related to the receipt by the Company of 69,899 shares of VectorY B.V.

in conjunction with a settlement agreement that the Company and VectorY B.V.

entered into in April 2021.

No such income was recorded in 2020 and 2019.

In 2021, 2020 and 2019 the Company’s other income also consisted of income from the subleasing of a portion of the Amsterdam facility while other expense consists of expenses incurred in relation to the subleasing income.

Income tax expense / (benefit)

Due to the uncertainty surrounding the realization of favorable tax attributes in future tax returns, the Company has recorded a valuation allowance against the Company’s net deferred tax assets in the Netherlands.

The Company released full valuation allowance against the Company’s net deferred tax assets in the United States as of December 31, 2020.

In connection with the Corlieve acquisition, the Company recognized a deferred tax liability related to acquired identifiable intangible assets and a deferred tax asset for net operating tax loss carryforwards for a net of EUR 11.9 million ($ 14.2 million) as of the Acquisition Date.

There are no significant unrecognized tax benefits as of December 31, 2021 and 2020.

For the years ended December 31, 2020, 2019 and 2018, loss before income taxes consists of the following:

For the years ended December 31, 2021, 2020 and 2019, income / (loss) before income tax (expense) / benefit consists of the following:

Years ended December 31,

1 unchanged sentence

Dutch operations

Foreign operations

The income tax benefit / (expense) for the years ended December 31, 2021, 2020 and 2019, consists of the following:

2 unchanged sentences

Current tax (expense)

Dutch operations

Foreign operations

Total current tax (expense)

Deferred tax benefit / (expense)

Deferred tax (expense) / benefit

Dutch operations

Foreign operations

Total deferred tax benefit / (expense)

Total income tax benefit / (expense)

Total deferred tax (expense) / benefit

Total income tax (expense) / benefit

Tax rate reconciliation

The reconciliation of the amount of income tax benefit / (expense) that would result from applying the Dutch statutory income tax rate to the Company’s reported amount of income tax benefit / (expense) for the years ended December 31, 2020, 2019 and 2018, is as follows:

The reconciliation of the amount of income tax (expense) / benefit that would result from applying the Dutch statutory income tax rate to the Company’s reported amount of income tax (expense) / benefit for the years ended December 31, 2021, 2020 and 2019, is as follows:

Years ended December 31,

(in thousands)

Loss before income tax income / (expense) for the period

Expected income tax benefit at the tax rate enacted in the Netherlands (25%)

Income / (loss) before income tax (expense) / benefit for the period

Expected income tax (expense) / benefit at the tax rate enacted in the Netherlands ( 25 %)

Non-deductible expenses

Other net change in valuation allowance

Difference in tax rates between the Netherlands and the U.S.

1 unchanged sentence

Release of valuation allowance related to expected future taxable income of U.S.

Other net change in valuation allowance

Nondeductible expenses

Change in fair value of contingent consideration

Income tax benefit / (expense)

Nondeductible expenses predominantly relate to share-based compensation expenses and affected the effective tax rate by an amount of $ 5.8 million in 2020 (2019:

Income tax (expense) / benefit

Non-deductible expenses predominantly relate to share-based compensation expenses and affected the effective tax rate by an amount of $ 6.7 million in 2021 (2020:

$ 5.8 million;

$ 4.4 million).

Derivative financial instruments affected the effective tax rate by income of $ 0.8 million in 2020 (expense of $ 0.6 million in 2019;

$ 0.0 million in 2018), which reduced the nondeductible expenses resulting from share-based compensation expenses.

The fair value loss on contingent consideration affected the effective tax rate by an amount of $ 2.0 million in 2021 ( nil in 2020 and 2019).

Significant components of deferred taxes

The tax effects of temporary differences and carryforwards that give rise to significant portions of deferred tax assets and deferred tax liabilities at December 31, 2020 and 2019 are as follows:

The tax effects of temporary differences and carryforwards that give rise to significant portions of deferred tax assets and deferred tax liabilities as of December 31, 2021 and 2020 are as follows:

Years ended December 31,

2 unchanged sentences

Net operating loss carryforwards

Lease liabilities

Operating lease liabilities

Intangible assets

Interest carryforwards

Accrued expenses and other current liabilities

Property, plant and equipment

Research and development tax credit carryforwards

Interest carryforwards

Derivative financial instrument

Deferred revenue

Gross deferred tax asset

Total deferred tax assets

Less valuation allowance

Net deferred tax asset

Right-of-use asset

Prepaid expenses

Deferred tax assets, net of valuation allowance

Acquired IPR&D Intangible Asset (see Note 3, "Corlieve transaction")

Operating lease right-of-use assets

Other current assets and receivables

Deferred tax liability

3 unchanged sentences

(in thousands)

Changes related to reduction of deferred revenue recorded in equity upon implementation of ASC 606 Revenue recognition as of January 1, 2018

Changes recorded in profit and loss

Increase/(reduction) related to 2020, 2019 and 2018 Dutch tax reforms

Changes recorded in the statement of operations

Increase related to 2021, 2020 and 2019 Dutch tax reforms

Valuation allowance assumed in Corlieve acquisition

Release of valuation allowance related to expected current year and future periods recorded in profit and loss

Other changes including currency translation effects

Included within changes recorded in profit and loss for the year ended December 31, 2020 are benefits of $ 1.2 million from the utilization of U.S.

net operating loss carryforwards ($ 0.8 million for the year ended December 31, 2019 and $ 0.9 million for the year ended December 31, 2018).

Other changes including currency translation adjustments

The Company released the full valuation allowance against the Company’s net deferred assets in the United States as of December 31, 2020.

Included within changes recorded in the statement of operations for the year ended December 31, 2020 and December 31, 2019 are benefits of $ 1.2 million and $ 0.8 million, respectively, from the utilization of U.S.

net operating loss carryforwards.

The valuation allowance as of December 31, 2021 is primarily related to net operating loss carryforwards in the Netherlands that, in the judgment of management, are not more-likely than-not to be realized.

In addition, the valuation allowance as of December 31, 2019 included deferred tax assets for net operating loss carryforwards and temporary differences in the United States of America.

Management considered projected future taxable income and tax-planning strategies in making this assessment.

A valuation allowance will be recorded against deferred tax assets if it is more likely than not that some or all the deferred tax assets will not be realized.

The Company determined that in accordance with Dutch tax law it would recognize the CSL Behring License Revenue as well as the License Fees as taxable results as of the date on which the Company is contractually entitled to receive (or obligated to make) a payment under the CSL Behring Agreement.

The Company expects to continue to incur taxable losses in the Netherlands except for the period in which it would receive the $ 450.0 million upfront payment under the CSL Behring Agreement.

In the event that the Company recognizes the $ 450.0 million upfront payment in 2021, such payment is expected to be subject to Dutch corporate income tax at a rate of 25.0 %.

However, the Company does not expect that it will be required to pay any income taxes in the period in which it recognizes the $ 450.0 million upfront payment as taxable revenue, as such payment is not expected to exceed the net operating losses that it carries forward in the Netherlands.

The Company specifically assessed the impact of the CSL Behring agreement with respect to retaining a full valuation allowance as of December 31, 2020.

Closing of the CSL Behring Agreement is contingent on the successful completion of reviews under the antitrust laws in the United States, Australia, and the United Kingdom.

The transactions have not closed as of December 31, 2020 as the Company received a Second Request from the United States Federal Trade Commission, on January 4, 2021.

Closing of the transaction is dependent on the timing, extent, and result of the Second Request.

In its assessment of whether or not it was more likely than not that the Company’s deferred tax assets in the Netherlands will be realized, the Company considered all relevant facts and circumstances, including similar regulatory reviews as well as the five-year cumulative losses reported by the Company in the Netherlands.

The Company concluded that it should continue to record a full valuation allowance as of December 31, 2020 in relation to its Dutch net operating loss carryforwards.

A portion of the valuation allowance for deferred tax assets relates to follow-on offering costs.

Management considered reversing taxable temporary differences, projected future taxable income and tax-planning strategies in making this assessment.

A valuation allowance was recorded against deferred tax assets if it is more likely than not that some or all the deferred tax assets will not be realized.

As of December 31, 2020, the Company had recorded a full valuation allowance against its Dutch net deferred tax assets.

On May 6, 2021, the CSL Behring Agreement became effective (refer to Note 4 “Collaboration arrangements and concentration of credit risk” ).

The Company recorded $ 462.4 million of license revenue related to closing the transaction.

The Company recorded such revenue in its Dutch tax return related to the 12-month period ended December 31, 2020, which it filed on February 10, 2022.

As such, the Company filed a return showing a taxable profit in the Netherlands in 2020, which resulted in the consumption of substantially all of its Dutch net operating losses for the years 2011 to 2018.

The Company’s remaining Dutch net operating tax losses carried forward relate to 2019 and 2021.

The Dutch government on June 4, 2021 enacted legislation, whereby such net operating tax losses can be carried forward indefinitely.

The Company expects to continue incurring tax losses for the foreseeable future.

As such, the Company retains its valuation allowance related to the deferred assets as of December 31, 2021.

The Company allocated the tax benefit from the release of the valuation allowance related to net operating loss carryforwards generated by share issuance cost incurred in 2014, 2015, 2017 and 2018 to additional paid-in capital.

This resulted in an increase of additional paid-in capital as well as deferred tax expenses of $ 3.0 million.

A portion of the valuation allowance for deferred tax assets relates to follow-on offering costs incurred in 2019 and costs related to the at-the-market offering in 2021.

Any subsequently recognized tax benefits will be credited directly to contributed capital.

As of December 31, 2021, that amount was $ 4.5 million ($ 7.7 million as of December 31, 2020).

The change is attributable to changes in the foreign currency rate.

The Dutch corporate tax rate for fiscal years 2019, 2020 and 2021 was 25 %.

During the years 2018 and 2019, the Dutch government enacted various changes to the corporate income tax rate applicable to future fiscal years.

In September 2020, further changes were enacted that retain the corporate rate at 25 % from 2021 onwards.

During 2019, the Dutch government enacted various changes to the corporate income tax rate applicable to future fiscal years.

In September 2020, further changes were enacted that retain the corporate income tax rate at 25 % from 2021 onwards.

In December 2021, even further changes were enacted that raised the corporate income tax rate from 25 % to 25.8 % from 2022 onwards.

A tax reform in December 2018 limited the carryforward of tax losses arising from January 1, 2019, to six years after the end of the respective period.

Tax losses incurred prior to this date continue to expire nine years after the end of the respective period.

As of December 31, 2020, new loss compensation rules were accepted by the Dutch Senate.

However, the bill includes a provision that the new compensation rules will be effective by a separate Governmental Decree, which has not been introduced as of December 31, 2020.

Hence, the new loss compensation rules are not enacted as of December 31, 2020.

If enacted, the rules allow losses to be carried forward indefinitely, but from fiscal year 2022 onwards would limit offsetting taxable profit in excess of EUR 1.0 million to 50% of the taxable profit.

In June 2021 legislation was enacted allowing for an indefinite carryforward from fiscal year 2022 onwards of existing and future net operating loss carryforwards subject to a limit of offsetting taxable profit in excess of EUR 1.0 million to 50% of the taxable profit.

The Dutch fiscal unity as of December 31, 2021 has an estimated $ 228.5 million (2020:

$ 588.2 million;

$ 311.7 million) of taxable losses that can be offset in the following six to seven years .

The expiration dates of these Dutch losses are summarized in the following table.

In the year ended December 31, 2020 unused tax losses of $ 18.5 million (December 31, 2019:

$ 20.7 million) expired.

(in thousands)

Loss expiring

The fiscal periods after 2017 are still open for inspection by the Dutch tax authorities.

$ 414.0 million) of taxable losses that are available for carry forward indefinitely.

In the year ended December 31, 2019, unused tax losses of $ 20.7 million expired.

The fiscal periods from 2019 onwards are still open for inspection by the Dutch tax authorities.

United States of America

The federal corporate tax rate in the U.S.

In addition, the Company is subject to state taxes resulting in a combined tax rate of 27.32 % for its U.S.

In addition, the Company is subject to state income taxes resulting in a combined tax rate of 27.32 % for its U.S.

As of December 31, 2021, an estimated $ 39.1 million of net operating losses remain to be carried forward.

1 unchanged sentence

The Company’s U.S.

operations generated taxable income in the fiscal years 2018, 2019 and 2020.

operations generated taxable income in the fiscal years 2018 to 2021.

Based on the current design of the Company’s worldwide operations, the Company expects to continue to generate taxable income in the U.S.

during the foreseeable future.

As of December 31, 2020, the Company considers it more likely than not that it will utilize its net deferred tax assets in the U.S.

and therefore released the remaining valuation allowance of $ 16.4 million through profit and loss as of this date.

Under the provision of the Internal Revenue Code, the U.S.

3 unchanged sentences

Subsequent ownership changes may further affect the limitation.

The fiscal periods after 2017 are still open for inspection by the Internal Revenue Service.

The fiscal periods from 2018 are still open for inspection by the Internal Revenue Service (“IRS”).

To the extent the Company has tax attribute carryforwards, the tax years in which the attribute was generated may still be adjusted upon examination by the IRS or Massachusetts Department of Revenue to the extent utilized in a future period.

The Company is currently not under examination by the IRS for any tax years.

The French corporate tax rate for fiscal years 2021 was 26.5 %, as of January 1, 2022 the tax rate is decreased to 25 %.

The Company’s French operation has incurred losses since incorporation and is expected to continue incurring tax losses for the foreseeable future.

The French operation as of December 31, 2021 has an estimated $ 9.1 million of taxable losses that are available for carry forward indefinitely.

Basic and diluted earnings per share

Diluted earnings per share is calculated by adjusting the weighted average number of ordinary shares outstanding, assuming conversion of all potentially dilutive ordinary shares.

As the Company has incurred a loss in the years presented, all potentially dilutive ordinary shares would have an antidilutive effect, if converted, and thus have been excluded from the computation of loss per share.

The shares are presented without giving effect to the application of the treasury method or exercise prices that would be above the share price as of December 31, 2020, December 31, 2019, and December 31, 2018, respectively.

In addition, the BMS warrants were not exercisable as of December 31, 2019, and December 31, 2018, since this would have required the prior designation of Collaboration Targets by BMS.

This would generally have resulted in a lower number of potentially dilutive ordinary shares as some stock option grants as well as the BMS warrants would have been excluded.

The potentially dilutive ordinary shares are summarized below:

Years ended December 31,

(ordinary shares)

BMS warrants (derecognized as of December 1, 2020 - see Note 4, "Fair value measurement")

Stock options under 2014 Plans

Non-vested RSUs and earned PSUs

Stock options under previous option plan

Hercules warrants (exercised February 1, 2019)

Basic net income / (loss) per ordinary share is computed by dividing net income / (loss) for the period by the weighted average number of ordinary shares outstanding during the period.

Diluted earnings per ordinary share are calculated by adjusting the weighted average number of ordinary shares outstanding, assuming conversion of all potentially dilutive ordinary shares.

For the year ended December 31, 2021, dilutive net income / (loss) per ordinary share is computed using the treasury method.

As the Company has incurred a loss in the years ended December 31, 2020 and December 31, 2019, all potentially dilutive ordinary shares for these years would have an antidilutive effect, if converted, and thus have been excluded from the computation of loss per share for the years ended December 31, 2020 and December 31, 2019.

Year ended December 31,

(in thousands)

Net income / (loss) attributable to ordinary shares

Weighted-average number of ordinary shares outstanding - basic

Stock options under 2014 Plans and previous plan

Non-vested RSUs and PSUs

Employee share purchase plan

Total potential dilutive ordinary shares

Weighted-average number of ordinary shares outstanding - diluted

The following table presents ordinary share equivalents that were excluded from the calculation of diluted net income / (loss) per ordinary share for the years ended December 31, 2021, 2020 and 2019 as the effect of their inclusion would have been anti-dilutive:

Year ended December 31,

(in thousands)

Anti-dilutive ordinary share equivalents

Stock options under 2014 Plans and previous plan

Non-vested RSUs and PSUs

Employee share purchase plan

BMS warrants (derecognized as of December 1, 2020 - refer to Note 5, "Fair value measurement"

Total anti-dilutive ordinary share equivalents

The anti-dilutive ordinary shares are presented without giving effect to the application of the treasury method or exercise prices that exceeded the price of the Company’s ordinary shares as of December 31, 2020 and December 31, 2019.

In addition, the BMS warrants were not exercisable as of December 31, 2019, since this would have required the prior designation of Collaboration Targets by BMS.

This would have resulted in a lower number of potentially dilutive ordinary shares as some stock option grants as well as the BMS warrants would have been excluded.

Commitments and contingencies

2 unchanged sentences

As both future sales levels and the timing and achievement of milestones are uncertain, the financial effect of these agreements cannot be estimated reliably.

The Company also has obligations to make future payments that become due and payable upon the collection of milestone payments from CSL Behring.

The achievement and timing of these milestones is not fixed and determinable.

Relevant commitments and contingencies are further discussed in other sections of this form 10-K, such as, Note 3 “ Corlieve transaction ” and Note 4 “ Collaboration arrangements and concentration of credit risk ”, amongst others.

Related party transaction

Between June 2015 and December 2020, BMS was considered a related party due to the combination of its equity investment in the Company (December 31, 2020:

2.4 million ordinary shares or 5.3 % of outstanding ordinary shares), the warrants as well as the potential obligations arising from the expansion of collaboration targets.

Between June 2015 and December 2020, BMS was considered a related party due to the combination of its equity investment in the Company, the warrants as well as the potential obligations arising from the expansion of collaboration targets.

On December 1, 2020, the Company entered into the amended BMS CLA.

All transactions subsequent to the effective date of the amended BMS CLA are considered to no longer be with a related party due to the elimination of the potential obligations related to additional Collaboration Targets (see Note 4 “Collaboration arrangements and concentration of credit risk” ) as well as the elimination of the BMS warrants (see Note 5, “Fair value measurement” ).

On October 21, 2021, the Company held an Extraordinary General Meeting of its shareholders and Rachelle Jacques was appointed to the Board of Directors (the “Board”) as a non-executive director.

Jacques will also serve as a member of the Audit Committee of the Board effective as of October 21, 2021.

On June 16, 2021, the Company’s shareholders voted to approve the reappointment of Mr.

David Meek and Ms.

Paula Soteropoulos as non-executive directors of the Board.

Meek has been appointed chairman of the Board.

Philip Astley-Sparke did not stand for reappointment and retired from the Board on June 16, 2021.

On June 15, 2021, Christian Klemt was appointed as Chief Financial Officer.

Klemt was our Chief Accounting Officer from August 2017 to June 2021, and he will continue to serve as general manager of our Amsterdam site.

Matthew Kapusta, who has been our Chief Executive Officer since December 2016 and had been our Chief Financial Officer from January 2015 to June 2021, will continue to serve as our Chief Executive Officer.

In connection with his transition to Chief Financial Officer, Mr.

Klemt will also serve as our Principal Financial Officer.

On May 17, 2021, Pierre Caloz was appointed as Chief Operating Officer.

Caloz oversees all manufacturing operations, global CMC development and innovation, supply chain, and facilities.

On September 14, 2020, the Company appointed Ricardo Dolmetsch, Ph.D.

13 unchanged sentences

Post has also assumed the role of chair of the Company’s Research and Development Committee of the Board of Directors.

On August 20, 2019, the Company promoted Alex Kuta, Ph.D., to Executive Vice President, Operations.

Kuta, in addition to regulatory affairs, became responsible for global quality as well as GMP manufacturing at the Company’s facility in Lexington, Massachusetts.

As of the same date Sander van Deventer, M.D., Ph.D., was promoted to Executive Vice President, Research and Product Development, and Dr.

van Deventer, in addition to his responsibilities for research, also became responsible for the Company’s product development.

As a result of these changes Scott McMillan, Ph.D.

retired from uniQure.

The employment of Dr.

van Deventer terminated on September 14, 2020.

In August 2019, the Company entered into an Amended and Restated Agreement Collaboration and License Agreement (“Amended CLA”) as well as an additional new Collaboration and License Agreement (“New CLA”) with its related party 4DMT Molecular Therapeutics, Inc.

In the Amended CLA, the Company received from 4DMT an exclusive, sublicensable, worldwide license under certain 4DMT intellectual property rights to research, develop, make, use, and commercialize previously selected Adeno-associated virus (“AAV”) capsid variants and certain associated products using 4DMT proprietary AAV technology for delivery of gene therapy constructs to cells in the central nervous system and the liver (“the Field”).

In the New CLA, the parties agreed to research and develop, at 4DMT’s cost, new AAV capsid variants using 4DMT proprietary AAV technology for delivery of up to six additional transgene constructs in the Field that will be selected by the Company.

As of June 2020, 4DMT is no longer considered a related party as a result of David Schaffer no longer being a non-executive member of the Company’s Board of Directors.

Subsequent events

Amendment Loan Facility

As of December 31, 2020, a $ 35.0 million term loan was outstanding in accordance with the 2018 Amended Facility between the Company and Hercules.

On January 29, 2021, the Company and Hercules amended the Company’s loan facility with Hercules Capital Inc.

entered, (“2021 Amended Facility”).

Pursuant to the 2021 Amended Facility, Hercules agreed to the 2021 Term Loan, increasing the aggregate principal amount of the term loan facility from $ 35.0 million to $ 135.0 million.

On January 29, 2021, the Company drew down $ 35.0 million of the 2021 Term Loan.

The Company may draw down the remaining $ 65.0 million under the 2021 Term Loan in a series of one or more advances of not less than $ 20.0 million each until December 15, 2021.

Advances under the 2021 Term Loan bear interest at a rate equal to the greater of (i) 8.25 % or (ii) 8.25 % plus the prime rate, less 3.25 % per annum.

The principal balance and all accrued but unpaid interest on advances under 2021 Term Loan is due on June 1, 2023, which date may be extended by the Company by up to two twelve-month periods.

Advances under the 2021 Term Loan may not be prepaid until six months after the Closing Date, following which the Company may prepay all such advances without charge.

In addition to the 2021 Term Loan, the amendment also extends the interest-only payment period of the previously funded $ 35.0 million term loan from January 1, 2022 to June 1, 2023.

The Company paid a $ 0.4 million facility charge on January 29, 2021.

End of term charges in respect of advances under the 2021 Term Loan range from 1.65 % to 6.85 % depending on the maturity date.

EXHIBIT INDEX

Amended Articles of Association of the Company (incorporated by reference to Exhibit 3.1 of the Company’s annual report on Form 10-K for the year ended December 31, 2018 (file no.

0001-36294) filed with the Securities and Exchange Commission).

Sale and Purchase Agreement, executed June 21, 2021, by and between uniQure N.V.

and Corlieve Therapeutics SAS (incorporated by reference to Exhibit 2.1 of the Company’s quarterly report on Form 10-Q (file no.

001-36294) for the period ending on June 30, 2021 filed with the Securities and Exchange Commission).

Amended Articles of Association of the Company (incorporated by reference to Exhibit 3.1 of the Company’s quarterly report on Form 10-Q (file no.

001-36294) for the period ending on June 30, 2021 filed with the Securities and Exchange Commission).

Description of the Registrant’s Securities Registered Pursuant to Section 12 of the Securities Exchange Act of 1934.

5 unchanged sentences

001-36294) for the period ending December 31, 2016 filed with the Securities and Exchange Commission).

Form of Restricted Stock Unit Award under the 2014 Share Incentive (incorporated by reference to Exhibit 10.4 of the Company's annual report on Form 10-K (file no.

001-36294) for the period ending December 31, 2017 filed with the Securities and Exchange Commission).

Form of Performance Stock Unit Award under the 2014 Share Incentive Plan (incorporated by reference to Exhibit 10.5 of the Company's annual report on Form 10-K (file no.

Form of Restricted Stock Unit Award under the 2014 Share Incentive Plan (incorporated by reference to Exhibit 10.4 of the Company's annual report on Form 10-K (file no.

001-36294) for the period ending December 31, 2017 filed with the Securities and Exchange Commission).

24 unchanged sentences

333-193158) filed with the Securities and Exchange Commission).

Second Amended and Restated Loan and Security Agreement, dated as of May 6, 2016 by and among uniQure Biopharma B.V., uniQure, Inc., uniQure IP B.V., the Company's subsidiaries listed therein, and Hercules Technology Growth Capital, Inc (incorporated by reference to Exhibit 10.30 of the Company's annual report on Form 10-K (file no.

001-36294) for the period ending December 31, 2016 filed with the Securities and Exchange Commission .

Collaboration and License Agreement by and between uniQure Biopharma B.V.

8 unchanged sentences

001-36294) for the period ending December 31, 2016 filed with the Securities and Exchange Commission).

Employment Agreement dated August 4, 2017 between uniQure biopharma B.V.

and Sander van Deventer (incorporated by reference to Exhibit 10.2 of the Company’s quarterly report on Form 10-Q (file no.

001-36294) for the period ending on June 30, 2017 filed with the Securities and Exchange Commission).

Employment Agreement dated July 15, 2017 between uniQure biopharma B.V.

7 unchanged sentences

0001-36294) filed with the Securities and Exchange commission).

Amendment No.

1 to Second Amended and Restated Loan and Security Agreement dates as of December 6, 2018 by and among uniQure Biopharma B.V., uniQure, Inc., uniQure IP B.V., the Company, and Hercules Technology Growth Capital, Inc (incorporated by reference to Exhibit 10.1 of the Company's current report on Form 8-K (file no.

001-36294) filed with the Securities and Exchange Commission) filed on December 10, 2018.

First Amendment Lease relating to 113 Hartwell Avenue, Lexington, Massachusetts, dated as of July 24, 2013, by and between the Company and King113 Hartwell LLC (incorporated by reference to Exhibit 10.1 of the Company's current report on form 8-K (file no.

12 unchanged sentences

001-36294) filed with the Securities and Exchange Commission) filed on September 20, 2019.

Amended and Restated Collaboration and License Agreement by and between 4D Molecular Therapeutics, Inc and uniQure biopharma B.V., dated August 6, 2019 (incorporated by reference to Exhibit 10.4 of the Company’s quarterly report on Form 10-Q (file no.

001-36294) for the period ending on September 30, 2019 filed with the Securities and Exchange Commission).

Collaboration and License Agreement by and between 4D Molecular Therapeutics, Inc and uniQure biopharma B.V., dates August 6, 2019 (incorporated by reference to Exhibit 10.5 of the Company’s quarterly report on Form 10-Q (file no.

001-36294) for the period ending on September 30, 2019 filed with the Securities and Exchange Commission).

Amended and Restated Employment Agreement, executed March 1, 2020 by and between uniQure biopharma B.V.

4 unchanged sentences

0001-36294) filed with the Securities and Exchange commission).

Amended and Restated Employment Agreement, executed March 1, 2020 by and between uniQure Inc.

and Maria Cantor (incorporated by reference to Exhibit 10.51 of the Company’s annual report on Form 10-K for the year ended December 31, 2019 (file no.

0001-36294) filed with the Securities and Exchange commission).

Amended and Restated Employment Agreement, executed March 1, 2020 by and between uniQure Inc.

and Jonathan Garen (incorporated by reference to Exhibit 10.52 of the Company’s annual report on Form 10-K for the year ended December 31, 2019 (file no.

0001-36294) filed with the Securities and Exchange commission).

Commercialization and License Agreement by and between uniQure biopharma B.V.

11 unchanged sentences

Amendment to Collaboration and License Agreement by and between uniQure biopharma B.V.

and Bristol-Myers Squibb Company dated December 1, 2020.

and Bristol-Myers Squibb Company dated December 1, 2020 (incorporated by reference to Exhibit 10.57 of the Company’s annual report on Form 10-K for the year ended December 31, 2020 (file no.

0001-36294) filed with the Securities and Exchange commission).

Amendment No.

2 to Second Amended and Restated Loan and Security Agreement as of January 29, 2021, by and among uniQure biopharma B.V., uniQure Inc., uniQure IP B.V., the Company and Hercules Capital Inc.

(incorporated by reference to Exhibit 10.58 of the Company’s annual report on Form 10-K for the year ended December 31, 2020 (file no.

0001-36294) filed with the Securities and Exchange commission).

Cooperation Agreement, dated as of April 16, 2021, by and among uniQure N.V., ForUniqure B.V., Forbion 1 Management B.V., Forbion International Management B.V., and Forbion Capital Partners Management Holding B.V.

(incorporated by reference to Exhibit 10.1 of the Company’s quarterly report on Form 10-Q (file no.

001-36294) for the period ending on March 31, 2021 filed with the Securities and Exchange Commission).

2014 Share Incentive Plan, Amended and Restated, effective as of June 16, 2021 (incorporated by reference to Exhibit 4.1 of the Company’s quarterly report on Form 10-Q (file no.

001-36294) for the period ending on June 30, 2021 filed with the Securities and Exchange Commission).

Employment Agreement, effective May 17, 2021, by and between uniQure biopharma B.V.

and Pierre Caloz (incorporated by reference to Exhibit 10.1 of the Company’s quarterly report on Form 10-Q (file no.

001-36294) for the period ending on June 30, 2021 filed with the Securities and Exchange Commission).

Equity Side Letter, effective May 17, 2021, by and between uniQure N.V.

and Pierre Caloz (incorporated by reference to Exhibit 10.2 of the Company’s quarterly report on Form 10-Q (file no.

001-36294) for the period ending on June 30, 2021 filed with the Securities and Exchange Commission).

Amended and Restated Employment Agreement, effective June 15, 2021, by and between uniQure biopharma B.V.

and Christian Klemt (incorporated by reference to Exhibit 10.3 of the Company’s quarterly report on Form 10-Q (file no.

001-36294) for the period ending on June 30, 2021 filed with the Securities and Exchange Commission).

Consent and Amendment No.

3 to Second Amended and Restated Loan and Security Agreement, dated July 30, 2021, by and among the Registrant, Hercules Capital Inc., and the other parties named therein (incorporated by reference to Exhibit 10.1 of the Company’s quarterly report on Form 10-Q (file no.

001-36294) for the period ending on September 30, 2021 filed with the Securities and Exchange Commission).

Form of Share Option Agreement, effective December 8, 2021, under the 2014 Share Incentive Plan.

Form of Restricted Stock Unit Award, effective December 8, 2021, under the 2014 Share Incentive Plan.

Form of Performance Stock Unit Award, effective December 8, 2021 under the 2014 Share Incentive Plan.

Third Amended and Restated Loan and Security Agreement as of December 15, 2021, by and among uniQure biopharma B.V., uniQure Inc., uniQure IP B.V., the Company and Hercules Capital Inc.

Lease Agreement relating to 20 Maguire Road, Lexington, Massachusetts, dated as of December 22, 2021, by and between uniQure Inc.

and G&I IX/GP4 20 Maguire LLC.

Lease Agreement relating to 91 Hartwell Avenue, Lexington, Massachusetts, dated as of February 1, 2022, by and between uniQure Inc.

and NRL 91 Hartwell LLC.

Code of Ethics (incorporated by reference to Exhibit 14.1 of the Company's annual report on Form 10-K (file no.

2 unchanged sentences

Consent of Independent Registered Public Accounting Firm – KPMG Accountants N.V.

Consent of Independent Registered Public Accounting Firm – PricewaterhouseCoopers Accountants N.V.

Power of Attorney (incorporated by reference to the signature page of this Annual Report on Form 10-K).

1 unchanged sentence

Rule 13a-14(a)/15d-14(a) Certification of Chief Financial Officer .

Section 1350 Certification .

Certification of Principal Executive Officer and Principal Financial Officer pursuant to 18 U.S.C.

Section 1350, as adopted pursuant to Section 906 of the Sarbanes-Oxley Act of 2002.

The following materials from the Company’s Annual Report on Form 10-K for the year ended December 31, 2021, formatted in Inline XBRL (eXtensible Business Reporting Language):

8 unchanged sentences

Matthew Kapusta

Chief Executive Officer

(Principal Executive and Financial Officer)

Chief Executive Officer (Principal Executive Officer)

/s/ CHRISTIAN KLEMT

Christian Klemt

Chief Financial Officer (Principal Financial Officer)

POWER OF ATTORNEY

2 unchanged sentences

/s/ MATTHEW KAPUSTA

Chief Executive Officer, Chief Financial Officer and Director (Principal Executive and Financial Officer)

March 1, 2021

Chief Executive Officer and Director (Principal Executive Officer)

February 25, 2022

Matthew Kapusta

/s/ CHRISTIAN KLEMT

Chief Accounting Officer

March 1, 2021

Chief Financial Officer (Principal Financial Officer)

February 25, 2022

Christian Klemt

/s/ PHILIP ASTLEY SPARKE

March 1, 2021

Philip Astley Sparke

/s/ MADHAVAN BALACHANDRAN

March 1, 2021

February 25, 2022

Madhavan Balachandran

/s/ ROBERT GUT

March 1, 2021

February 25, 2022

/s/ RACHELLE JACQUES

February 25, 2022

Rachelle Jacques

/s/ JACK KAYE

March 1, 2021

February 25, 2022

/s/ DAVID MEEK

March 1, 2021

February 25, 2022

/s/ LEONARD POST

March 1, 2021

February 25, 2022

/s/ PAULA SOTEROPOULOS

March 1, 2021

February 25, 2022

Paula Soteropoulos

/s/ JEREMY P.

March 1, 2021

February 25, 2022

Compared sentence by sentence after normalising whitespace, quotation marks, case and digits, so re-formatting and restated figures do not read as changed language. Wording changes appear as one removal and one addition. The current filing and the prior one are authoritative.

What changed 10-K

Item 9A. Controls and Procedures