PART I – FINANCIAL INFORMATION [Financial Statements](index=5&type=section&id=Item%201.%20Financial%20Statements) The company reported **$8.5 million** in Q1 2024 revenues and a **$65.6 million** net loss, with **$770.1 million** total assets and **$146.8 million** equity as of March 31, 2024 Consolidated Balance Sheet Summary (Unaudited) | Balance Sheet Items | March 31, 2024 (in thousands) | December 31, 2023 (in thousands) | | :--- | :--- | :--- | | **Assets** | | | | Cash and cash equivalents | $243,062 | $241,360 | | Current investment securities | $312,621 | $376,532 | | Total current assets | $596,003 | $651,853 | | Total assets | $770,050 | $831,689 | | **Liabilities & Equity** | | | | Total current liabilities | $63,473 | $73,675 | | Long-term debt | $102,120 | $101,749 | | Liability from royalty financing agreement | $405,398 | $394,241 | | Total liabilities | $623,296 | $624,019 | | Total shareholders' equity | $146,754 | $207,670 | Consolidated Statement of Operations Summary (Unaudited) | Income Statement Items | Three months ended March 31, 2024 (in thousands) | Three months ended March 31, 2023 (in thousands) | | :--- | :--- | :--- | | Total revenues | $8,485 | $5,325 | | Research and development expenses | ($40,692) | ($60,809) | | Selling, general and administrative expenses | ($13,937) | ($17,848) | | Loss from operations | ($54,228) | ($74,172) | | Net loss | ($65,618) | ($77,227) | | Basic and diluted net loss per ordinary share | ($1.36) | ($1.63) | Consolidated Statement of Cash Flows Summary (Unaudited) | Cash Flow Items | Three months ended March 31, 2024 (in thousands) | Three months ended March 31, 2023 (in thousands) | | :--- | :--- | :--- | | Net cash used in operating activities | ($60,575) | ($78,302) | | Net cash generated from investing activities | $63,985 | $2,988 | | Net cash generated from financing activities | $0 | $131 | | Net increase / (decrease) in cash | $1,685 | ($74,149) | [Management's Discussion and Analysis of Financial Condition and Results of Operations](index=30&type=section&id=Item%202.%20Management%27s%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management discusses the company's gene therapy pipeline, Q1 2024 financial performance with increased revenues and reduced net loss, and projected cash runway into Q2 2027 - uniQure is a gene therapy company with one approved product, **HEMGENIX®** for **hemophilia B**, and a pipeline including **AMT-130** for **Huntington's disease**, **AMT-162** for **ALS**, **AMT-260** for **refractory MTLE**, and **AMT-191** for **Fabry disease**[87](index=87&type=chunk) - As of March 31, 2024, the company held **$555.7 million** in cash, cash equivalents, and investment securities, projected to fund operations into the **second quarter of 2027**[101](index=101&type=chunk)[139](index=139&type=chunk) Financial Highlights Q1 2024 vs Q1 2023 | Metric | Q1 2024 (in thousands) | Q1 2023 (in thousands) | Change | | :--- | :--- | :--- | :--- | | Total Revenues | $8,485 | $5,325 | $3,160 | | R&D Expenses | ($40,692) | ($60,809) | $20,117 | | SG&A Expenses | ($13,937) | ($17,848) | $3,911 | | Net Loss | ($65,618) | ($77,227) | $11,609 | - R&D expenses decreased by **$20.1 million** to **$40.7 million** in Q1 2024, driven by a **$13.3 million** reduction in direct program spending, especially for **AMT-162 (ALS)**, and lower personnel costs post-2023 restructuring[120](index=120&type=chunk)[121](index=121&type=chunk)[131](index=131&type=chunk) - Interest expense increased to **$16.1 million** in Q1 2024 from **$3.6 million** in Q1 2023, primarily due to **$12.4 million** in interest expense from the May 2023 Royalty Financing Agreement[135](index=135&type=chunk) [Quantitative and Qualitative Disclosures About Market Risk](index=51&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) The company's market risks, including currency, price, interest rate, credit, and liquidity risks, remained materially unchanged in Q1 2024 - The company's market risks, encompassing currency, price, interest rate, credit, and liquidity risks, have not materially changed during Q1 2024[164](index=164&type=chunk)[165](index=165&type=chunk) [Controls and Procedures](index=51&type=section&id=Item%204.%20Controls%20and%20Procedures) Management concluded the company's disclosure controls and procedures were effective as of March 31, 2024, with no material changes in internal control over financial reporting during the quarter - The CEO and CFO concluded that the company's disclosure controls and procedures were **effective** as of March 31, 2024[166](index=166&type=chunk) - No material changes occurred in internal control over financial reporting during Q1 2024[167](index=167&type=chunk) PART II – OTHER INFORMATION [Legal Proceedings](index=52&type=section&id=Item%201.%20Legal%20Proceedings) The company reported no legal proceedings during the period - The company reports no legal proceedings to disclose for the period[170](index=170&type=chunk) [Risk Factors](index=52&type=section&id=Item%201A.%20Risk%20Factors) The company faces significant risks including dependency on **AMT-130** clinical success, manufacturing complexities, funding needs, regulatory hurdles, and stock price volatility - The company's business is highly dependent on the clinical success of its lead candidate, **AMT-130** for **Huntington's disease**[173](index=173&type=chunk)[177](index=177&type=chunk) - Gene therapies are complex and expensive to manufacture, posing potential production challenges that could delay development and commercialization[173](index=173&type=chunk)[224](index=224&type=chunk) - The company has a history of net losses and requires additional funding to advance product candidates; failure to secure capital could delay or terminate development[173](index=173&type=chunk)[332](index=332&type=chunk) - Reliance on third parties for clinical trials introduces risks of delays or termination due to poor performance, impacting commercial prospects[180](index=180&type=chunk)[279](index=279&type=chunk) - The company's stock price remains volatile, influenced by clinical trial results, regulatory developments, and market conditions[173](index=173&type=chunk)[379](index=379&type=chunk) - The October 2023 restructuring, aimed at portfolio reprioritization and resource conservation, carries risks such as unachieved benefits, employee attrition, and management distraction[375](index=375&type=chunk)[377](index=377&type=chunk) [Unregistered Sales of Equity Securities, Use of Proceeds, and Issuer Purchases of Equity Securities](index=128&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%2C%20Use%20of%20Proceeds%2C%20and%20Issuer%20Purchases%20of%20Equity%20Securities) The company reported no unregistered sales of equity securities, use of proceeds, or issuer purchases during the period - None reported[403](index=403&type=chunk) [Other Information](index=128&type=section&id=Item%205.%20Other%20Information) No director or officer adopted or terminated a Rule 10b5-1 or non-Rule 10b5-1 trading arrangement in Q1 2024 - No director or officer adopted or terminated a Rule 10b5-1 or non-Rule 10b5-1 trading arrangement in Q1 2024[406](index=406&type=chunk) [Exhibits](index=128&type=section&id=Item%206.%20Exhibits) This section lists exhibits filed with the Form 10-Q, including CEO and CFO certifications and iXBRL financial statements - The report includes required certifications from the CEO and CFO as exhibits[410](index=410&type=chunk)

Exhibit 99.1 uniQure Announces 2023 Financial Results and Highlights Recent Company Progress ~ Presented promising clinical update from U.S. and European Phase I/II trials of AMT-130 in Huntington's disease; Up to three years of follow-up data to be presented in mid-2024; Regulatory interactions and clarity on potential strategies for clinical development expected in 2024 ~ ~ Announced FDA clearance of two Investigational New Drug (IND) applications; Initiation of Phase I/II clinical trials in mesial tempor ...

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington D.C. 20549 FORM 10-K (Mark One) ☒ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended December 31, 2023 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission file number: 001-36294 uniQure N.V. (Exact name of Registrant as specified in its charter) The Netherlands (Jurisdiction of incorpor ...

[PART I – FINANCIAL INFORMATION](index=5&type=section&id=PART%20I%20%E2%80%93%20FINANCIAL%20INFORMATION) [Financial Statements](index=5&type=section&id=Item%201.%20Financial%20Statements) The unaudited consolidated financial statements for uniQure N.V. as of September 30, 2023, show increased total assets and a widened net loss due to higher expenses and a new royalty financing liability Consolidated Balance Sheet Highlights (in thousands) | Balance Sheet Items | Sep 30, 2023 (in thousands) | Dec 31, 2022 (in thousands) | | :--- | :--- | :--- | | Cash and cash equivalents | $229,484 | $228,012 | | Total current assets | $693,626 | $476,774 | | Total assets | $871,550 | $704,964 | | Total current liabilities | $68,607 | $75,919 | | Liability from royalty financing agreement | $383,711 | - | | Total liabilities | $602,766 | $228,955 | | Total shareholders' equity | $268,784 | $476,009 | Consolidated Statements of Operations Highlights (in thousands) | Income Statement Items | Nine months ended Sep 30, 2023 (in thousands) | Nine months ended Sep 30, 2022 (in thousands) | | :--- | :--- | :--- | | Total revenues | $9,154 | $3,738 | | Research and development expenses | ($172,245) | ($139,263) | | Selling, general and administrative expenses | ($57,103) | ($36,802) | | Loss from operations | ($221,123) | ($169,660) | | Interest expense | ($25,846) | ($8,279) | | Net loss | ($235,272) | ($133,596) | | Net loss per ordinary share | ($4.94) | ($2.86) | Consolidated Statements of Cash Flows Highlights (in thousands) | Cash Flow Items | Nine months ended Sep 30, 2023 (in thousands) | Nine months ended Sep 30, 2022 (in thousands) | | :--- | :--- | :--- | | Net cash used in operating activities | ($96,388) | ($90,380) | | Net cash used in investing activities | ($265,248) | ($14,522) | | Net cash generated from financing activities | $362,675 | $844 | | Net increase / (decrease) in cash | $1,463 | ($115,934) | - In May 2023, the company entered into a **Royalty Financing Agreement**, selling certain royalty rights on **HEMGENIX®** net sales to HemB SPV for an upfront payment, accounted for as debt[36](index=36&type=chunk) - On October 5, 2023, the company announced a reorganization plan to reduce its global workforce by approximately **20%** and discontinue over half of its research and technology projects, with an estimated cost of approximately **$2.3 million** in employee severance[86](index=86&type=chunk) [Management's Discussion and Analysis of Financial Condition and Results of Operations](index=31&type=section&id=Item%202.%20Management%27s%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management discusses the company's gene therapy leadership, strategic reorganization, key clinical program advancements, and financial performance, including a widened net loss and extended cash runway [Business Developments](index=31&type=section&id=Business%20Developments) The company underwent a major reorganization to prioritize clinical programs like AMT-130, AMT-260, AMT-162, and AMT-191, secured $375 million from a royalty financing agreement, and received FDA clearance for AMT-260 IND - The company announced a reorganization to eliminate **~20%** of its workforce, close a research lab, and discontinue over half of its research and technology projects to prioritize advancing clinical-stage programs, with expected costs of **~$2.3 million** for severance[91](index=91&type=chunk)[92](index=92&type=chunk) - The FDA cleared the Investigational New Drug (IND) application for **AMT-260**, a gene therapy candidate for refractory mesial temporal lobe epilepsy (MTLE), in **August 2023**[89](index=89&type=chunk)[95](index=95&type=chunk) - Interim data from the Phase I/II trial of **AMT-130** for Huntington's disease showed the treatment was generally well-tolerated with a manageable safety profile, and clinical function was generally preserved at **24 months** (low-dose) and **12 months** (high-dose) compared to baseline[100](index=100&type=chunk)[101](index=101&type=chunk)[103](index=103&type=chunk) - The company entered into a royalty financing agreement, receiving an upfront payment of **$375.0 million** in exchange for rights to the lowest royalty tier on **HEMGENIX®** worldwide net sales[114](index=114&type=chunk) [Results of Operations](index=43&type=section&id=Results%20of%20Operations) Total revenues increased to $9.2 million, but net loss widened to $235.3 million due to higher R&D expenses, increased SG&A costs, and a surge in interest expense from the new royalty financing agreement Comparison of Operations (Nine Months Ended Sep 30, in thousands) | Line Item | 2023 (in thousands) | 2022 (in thousands) | Change (in thousands) | | :--- | :--- | :--- | :--- | | Total revenues | $9,154 | $3,738 | $5,416 | | R&D expenses | ($172,245) | ($139,263) | ($32,982) | | SG&A expenses | ($57,103) | ($36,802) | ($20,301) | | Loss from operations | ($221,123) | ($169,660) | ($51,463) | | Net loss | ($235,272) | ($133,596) | ($101,676) | Revenue Breakdown (Nine Months Ended Sep 30, in thousands) | Revenue Source | 2023 (in thousands) | 2022 (in thousands) | Change (in thousands) | | :--- | :--- | :--- | :--- | | License revenues | $1,290 | $0 | $1,290 | | Contract manufacturing revenues | $6,596 | $0 | $6,596 | | Collaboration revenues | $1,268 | $3,738 | ($2,470) | | **Total revenues** | **$9,154** | **$3,738** | **$5,416** | Direct R&D Expenses by Program (Nine Months Ended Sep 30, in thousands) | Program | 2023 (in thousands) | 2022 (in thousands) | Change (in thousands) | | :--- | :--- | :--- | :--- | | Amyotrophic lateral sclerosis (AMT-162) | $13,048 | $0 | $13,048 | | Temporal lobe epilepsy (AMT-260) | $11,970 | $11,394 | $576 | | Huntington's disease (AMT-130) | $11,093 | $15,721 | ($4,628) | | Fabry disease (AMT-191) | $1,846 | $1,664 | $182 | [Financial Position, Liquidity and Capital Resources](index=58&type=section&id=Financial%20Position%2C%20Liquidity%20and%20Capital%20Resources) As of September 30, 2023, the company held $662.1 million in liquid assets, projecting funding into Q2 2027, supported by royalty financing proceeds and a milestone payment, while managing significant debt and lease obligations - The company believes its cash, cash equivalents, and investment securities of **$662.1 million** as of September 30, 2023, will fund operations into the **second quarter of 2027**[187](index=187&type=chunk) - Net cash used in operating activities was **$96.4 million** for the nine months ended Sep 30, 2023, compared to **$90.4 million** in the prior year period[202](index=202&type=chunk) - Net cash generated from financing activities was **$362.7 million**, primarily due to **$370.1 million** in net proceeds from the **Royalty Financing Agreement**[208](index=208&type=chunk)[209](index=209&type=chunk) - Key contractual obligations include **$100.0 million** in principal debt to Hercules due in **2027**, the royalty financing agreement liability, **$54.8 million** in lease payments, and up to **EUR 190.0 million** in potential milestone payments related to the uniQure France SAS acquisition[188](index=188&type=chunk)[189](index=189&type=chunk)[191](index=191&type=chunk)[192](index=192&type=chunk) [Quantitative and Qualitative Disclosures About Market Risk](index=67&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) The company's market risks, including currency, price, and interest rate fluctuations, remain consistent with prior disclosures, with a focus on capital preservation and minimizing adverse financial market effects - The company's market risks, including currency, price, and interest rate risk, have **not materially changed** during the nine months ended September 30, 2023, from what was disclosed in the Annual Report[218](index=218&type=chunk) [Controls and Procedures](index=67&type=section&id=Item%204.%20Controls%20and%20Procedures) Management concluded that disclosure controls and procedures were effective as of September 30, 2023, with no material changes to internal control over financial reporting during the quarter - Based on an evaluation as of September 30, 2023, the CEO and CFO concluded that the company's disclosure controls and procedures were **effective**[219](index=219&type=chunk) - There were **no changes** in the company's internal control over financial reporting during the quarter that materially affected, or are reasonably likely to materially affect, internal controls[220](index=220&type=chunk) [PART II – OTHER INFORMATION](index=68&type=section&id=PART%20II%20%E2%80%93%20OTHER%20INFORMATION) [Legal Proceedings](index=68&type=section&id=Item%201.%20Legal%20Proceedings) The company reported no legal proceedings during the period - None[223](index=223&type=chunk) [Risk Factors](index=68&type=section&id=Item%201A.%20Risk%20Factors) The company faces significant risks including clinical trial delays, financial funding needs, operational dependencies, strategic reorganization effectiveness, and intellectual property protection challenges - Clinical Trial Risks: The company has encountered and may continue to encounter **delays** in clinical trials or **fail to demonstrate the safety and efficacy** of its product candidates[226](index=226&type=chunk) - Financial Risks: The company will likely need to raise **additional funding**, which may **not be available**, and the amount required depends on future payments from CSL Behring for **HEMGENIX®**[226](index=226&type=chunk) - Operational Risks: The company's manufacturing facility is subject to **significant government regulations**, and it also **relies on third parties** for preclinical studies and clinical trials, who may **not perform satisfactorily**[226](index=226&type=chunk) - Strategic Risks: Actions taken to restructure the business, including the recent Reorganization, may **not be as effective as anticipated**, and the company may also **not be successful** in its efforts to build a pipeline of additional product candidates[226](index=226&type=chunk)[227](index=227&type=chunk) - Intellectual Property Risks: The company's ability to commercialize products may be **impaired** if it is **unable to obtain and maintain patent protection** for its technology and products[226](index=226&type=chunk) [Unregistered Sales of Equity Securities, Use of Proceeds, and Issuer Purchases of Equity Securities](index=131&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%2C%20Use%20of%20Proceeds%2C%20and%20Issuer%20Purchases%20of%20Equity%20Securities) The company reported no unregistered sales of equity securities, use of proceeds, or issuer purchases of equity securities during the period - None[434](index=434&type=chunk) [Defaults Upon Senior Securities](index=131&type=section&id=Item%203.%20Defaults%20Upon%20Senior%20Securities) The company reported no defaults upon senior securities - None[435](index=435&type=chunk) [Mine Safety Disclosures](index=131&type=section&id=Item%204.%20Mine%20Safety%20Disclosures) This item is not applicable to the company - Not applicable[436](index=436&type=chunk) [Other Information](index=131&type=section&id=Item%205.%20Other%20Information) No director or officer adopted or terminated a Rule 10b5-1 or non-Rule 10b5-1 trading arrangement during the third quarter of 2023 - **No director or officer adopted or terminated** a Rule 10b5-1 trading arrangement or non-Rule 10b5-1 trading arrangement during the three months ended September 30, 2023[437](index=437&type=chunk) [Exhibits](index=131&type=section&id=Item%206.%20Exhibits) This section lists filed exhibits, including the Termination and Consulting Agreement with Dr. Ricardo Dolmetsch, CEO and CFO certifications, and iXBRL data files - Filed exhibits include **CEO and CFO certifications** (31.1, 31.2, 32.1) and the **Termination and Consulting Agreement** for Ricardo Dolmetsch, Ph.D. (10.1)[441](index=441&type=chunk)

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington D.C. 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended June 30, 2023 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from _______ to _______ Commission file number: 001-36294 uniQure N.V. (Exact name of Registrant as specified in its charter) The Netherlands (Stat ...

Financial Performance - Total revenues for Q1 2023 were $5.325 million, a significant increase from $1.792 million in Q1 2022, primarily driven by contract manufacturing revenues of $4.937 million[17]. - The net loss for Q1 2023 was $77.227 million, compared to a net loss of $46.678 million in Q1 2022, indicating a worsening financial performance year-over-year[17]. - Cash used in operating activities for Q1 2023 was $78.302 million, significantly higher than $25.711 million in Q1 2022[22]. - The company reported a basic and diluted net loss per ordinary share of $1.63 for Q1 2023, compared to $1.00 for Q1 2022[17]. - The accumulated deficit as of March 31, 2023, was $659.2 million, up from $581.9 million as of December 31, 2022[85]. - The company reported a net loss of $77.2 million in Q1 2023, compared to a net loss of $46.7 million in Q1 2022, representing a 65.2% increase in losses[132]. Cash and Assets - Cash and cash equivalents decreased to $153.851 million as of March 31, 2023, down from $228.012 million at the end of 2022[14]. - Total assets decreased to $631.004 million as of March 31, 2023, from $704.964 million at the end of 2022[14]. - Total shareholders' equity fell to $412.771 million as of March 31, 2023, down from $476.009 million at the end of 2022[14]. - The company had cash and cash equivalents of $153.851 million as of March 31, 2023, down from $228.012 million as of December 31, 2022[42]. - As of March 31, 2023, cash and cash equivalents totaled $318.5 million, with expectations to fund operations into 2025[125]. Research and Development - Research and development expenses rose to $60.809 million in Q1 2023, compared to $45.003 million in Q1 2022, reflecting increased investment in product development[17]. - The company anticipates increased expenses related to advancing AMT-130 into phase III clinical studies and other gene therapy programs[86]. - The company incurred $4.3 million and $2.6 million for the preclinical development of temporal lobe epilepsy in Q1 2023 and Q1 2022, respectively, reflecting a 65.4% increase[110]. - The total research and development expenses for Q1 2023 were $18.7 million, compared to $16.2 million in Q1 2022, marking a 15.4% increase[115]. - The company spent $1.9 million on preclinical programs in Q1 2023, up from $1.3 million in Q1 2022, indicating a 46.2% increase[112]. Clinical Trials and Product Development - The company is conducting a Phase I/II clinical trial for AMT-130, its gene therapy candidate for Huntington's disease, with 26 patients enrolled[79]. - AMT-130 showed a 53.8% decline in mutant Huntingtin protein levels in cerebral spinal fluid at 12 months of follow-up in treated patients[82]. - The company initiated a GLP toxicology study for AMT-260 in the third quarter of 2022, targeting refractory temporal lobe epilepsy[83]. - The company has a pipeline of innovative gene therapies, including candidates for Huntington's disease and ALS, with HEMGENIX® approved for commercialization in the U.S. and Europe[75]. Revenue Sources - Contract manufacturing revenues recognized for HEMGENIX® amounted to $4.9 million in the three months ended March 31, 2023, compared to nil in the same period in 2022[104]. - Collaboration revenues decreased to $0.4 million in the three months ended March 31, 2023, from $1.4 million in the same period in 2022[103]. Expenses and Liabilities - Selling, general and administrative expenses rose to $17.8 million in Q1 2023 from $11.0 million in Q1 2022, a significant increase of 61.8%[117]. - Interest expense associated with the 2021 Restated Facility was $3.6 million for the three months ended March 31, 2023, compared to $2.4 million during the same period in 2022[54]. - The company had total liabilities of $37.222 million as of March 31, 2023, which included $36.894 million in contingent consideration[42]. Regulatory and Compliance Risks - The company faces significant risks related to FDA compliance and manufacturing regulations, which could materially harm its business if not adhered to[190]. - The complexity of gene therapies may lead to production difficulties, impacting the ability to meet regulatory requirements and commercial prospects[194]. - Regulatory approval processes for gene therapies are expensive and lengthy, with potential for significant delays or rejection of applications[200]. - The company has encountered risks related to the compliance of its manufacturing facility with government regulations, which could materially harm its business[158]. Future Outlook - Future capital requirements will depend on various factors, including milestone payments from CSL Behring and the costs of clinical trials for AMT-130[145]. - The company has potential milestone payments of up to $43.5 million related to the Corlieve acquisition, with additional payments of $174.0 million tied to Phase III development[128].

Charles, Huntington's disease patient advocate LE A D E R S H IP IN GE N E T H E R A P Y LEADERSHIP IN GENE THERAPY MARCH 2023 MA R C H 2023 | 22 First approved AAV gene therapy First AAV vector demonstrated effective in patients with NABs MARCH 2023 a case study in delivering value through innovation 2017 Announced transition to AMT-061 2018 Initiated Ph 2b doseconfirmation study and HOPE-B pivotal study leading the gene therapy revolution • Broad pipeline of first/best-in-class gene therapies • EU approva ...

Financial Milestones - The company reported a significant milestone payment of $75.0 million from CSL Behring upon the first sale of HEMGENIXÔ in the European Union prior to July 2, 2023[14]. - The company may need to raise additional funding, which could be affected by a $75.0 million milestone payment related to HEMGENIXÔ sales in the EU[14]. Clinical Trials and Product Development - The company anticipates continued reliance on third parties for conducting clinical trials, which may impact timelines and regulatory compliance[14]. - The company has encountered delays in clinical trials and may continue to face challenges in demonstrating the safety and efficacy of its product candidates[14]. - The company may not successfully build a pipeline of additional product candidates using its gene therapy technology platform[14]. - The company is focused on building a pipeline of additional product candidates using its gene therapy technology platform[14]. - The company is reliant on third parties for conducting clinical trials, which may not perform satisfactorily or meet regulatory requirements[14]. Regulatory and Compliance Risks - The company is subject to significant government regulations regarding its manufacturing facility, and failure to comply could harm its business[14]. - The company must comply with complex data protection laws, and failure to do so may result in penalties[14]. - The company is exposed to risks associated with data protection laws across different jurisdictions, with potential penalties for non-compliance[14]. Operational Challenges - The ongoing Covid pandemic has materially affected the company's operations and supply chain[15]. - The ongoing Covid pandemic has materially affected the company's business, operations, and supply chain[15]. - The company faces risks related to competition, pricing, and reimbursement for its product candidates, which could adversely affect revenue[14]. - The company faces competition and challenges in employee recruitment and retention, which could adversely affect its business[14]. Internal Controls and Security - The company emphasizes the importance of maintaining effective internal controls to ensure accurate reporting and prevent fraud[15]. - The company’s internal computer systems may suffer security breaches or disruptions, impacting product development programs[14]. Funding and Intellectual Property - The company may need to raise additional funding, which may not be available on acceptable terms, potentially impacting product development efforts[14]. - The company relies on licenses of intellectual property from third parties, which may not be available in the future on commercially reasonable terms[14]. - The company relies on licenses of intellectual property from third parties, which may not be available on commercially reasonable terms[14].

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington D.C. 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended September 30, 2022 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from _______ to _______ Commission file number: 001-36294 uniQure N.V. (Exact name of Registrant as specified in its charter) The Netherlands (State or other ju ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington D.C. 20549 ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 FORM 10-Q For the transition period from _______ to _______ (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended June 30, 2022 OR Commission file number: 001-36294 uniQure N.V. (Exact name of Registrant as specified in its charter) The Netherlands (State or other jurisdi ...