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 ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION uniQure N.V. (Exact name of Registrant as specified in its charter) 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 March 31, 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 The Netherlands (State or other jurisd ...

COVID-19 Impact - The company reported a significant impact from the ongoing COVID-19 pandemic on its business and operations[16] - The ongoing Covid pandemic has materially and adversely affected the company's business, operations, and supply chain[16] Clinical Trials and Product Development - There are delays and challenges in the progress of clinical trials, which may affect the safety and efficacy demonstration of product candidates[16] - The company has encountered delays in clinical trials and may fail to demonstrate the safety and efficacy of its product candidates[16] - There is uncertainty regarding the successful commercialization of product candidates and potential delays could materially harm the business[16] - The company relies on third parties for clinical trials, which may not perform satisfactorily, impacting timelines and regulatory compliance[16] Regulatory Compliance - Regulatory compliance is critical for the manufacturing facility, and failure to maintain approvals could materially harm the business[16] - The company is subject to significant government regulations and approvals for its manufacturing facility, and failure to comply could materially harm the business[16] - Data protection laws in various jurisdictions pose compliance challenges, and failure to comply may result in penalties affecting the business[19] - The company faces potential penalties for non-compliance with data protection laws, which could adversely affect its financial condition[19] Funding and Financial Challenges - The company may need to raise additional funding, which could be challenging and affect product development efforts[16] - The company may need to raise additional funding, which may not be available on acceptable terms, potentially affecting product development efforts[16] Competition and Market Risks - The company is exposed to competition and pricing pressures that could adversely affect product revenue[16] - The company faces competition and challenges in recruiting and retaining employees, which could adversely affect its operations[16] Intellectual Property Risks - Intellectual property licensing from third parties may not be available on commercially reasonable terms, impacting product commercialization[16] - The company relies on licenses of intellectual property from third parties, which may not be available in the future on commercially reasonable terms[16] Internal Controls and Security - Internal control failures could lead to inaccurate reporting and negatively impact investor confidence and share price[19] - Internal computer systems may suffer security breaches or disruptions, potentially disrupting product development programs[19]

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, 2021 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 ...

| --- | --- | --- | --- | --- | --- | --- | --- | |-------|-------|-------|-------|-------|-------|-------|----------------------------------------| | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | Leadership in | | | | | | | | | Gene Therapy | | | | | | | | | Corporate Presentation September 2021 | Forward-looking Statements This presentation contains forward-looking statements. All statements other than statements of historical fact are forward- looking statements, w ...

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, 2021 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 jurisdi ...

Financial Performance - Total revenues for Q1 2021 were $454,000, a decrease of 56.3% compared to $1,040,000 in Q1 2020[17] - Net loss for Q1 2021 was $41,556,000, compared to a net loss of $27,999,000 in Q1 2020, representing a 48.3% increase in losses[17] - Basic and diluted net loss per ordinary share for Q1 2021 was $0.91, compared to $0.63 in Q1 2020[17] - Total revenues for the three months ended March 31, 2021, were $454,000, compared to $104,000 for the same period in 2020, representing a 336% increase[127] - Collaboration revenue increased to $454,000 in Q1 2021 from $57,000 in Q1 2020, a growth of 696%[128] - The net loss for the three months ended March 31, 2021, was $41.6 million, up from a net loss of $28.0 million in the same period of 2020, indicating higher operational costs[107] Expenses - Research and development expenses increased to $32,656,000 in Q1 2021, up 25.5% from $26,013,000 in Q1 2020[17] - Selling, general and administrative expenses increased to $12.4 million in Q1 2021 from $9.1 million in Q1 2020, reflecting a 36.3% rise[139] - Personnel and contractor-related expenses increased to $11.6 million in Q1 2021 from $9.3 million in Q1 2020, a rise of 24.6%[142] - The company incurred $2.7 million in share-based compensation expenses in Q1 2021, compared to $2.4 million in Q1 2020, marking a 12.5% increase[142] - Share-based compensation expense for the three months ended March 31, 2021 was $5.754 million, an increase from $4.340 million in the same period in 2020[62] Cash and Liquidity - Cash and cash equivalents at the end of Q1 2021 were $260,813,000, an increase of 6.3% from $244,932,000 at the end of 2020[14] - As of March 31, 2021, cash and cash equivalents totaled $260.8 million, an increase from $244.9 million as of December 31, 2020[107] - The company expects cash and cash equivalents, along with a $100 million facility, to fund operations until the end of 2022, with sufficient funds projected into the second half of 2024 following a $450 million payment received on May 7, 2021[146] - The company expects to fund its operating expenses and capital expenditure requirements into the second half of 2024 with existing cash and the $100.0 million 2021 Amended Facility[163] Assets and Liabilities - Total assets as of March 31, 2021, were $363,853,000, up 6.9% from $340,395,000 at the end of 2020[14] - Total liabilities increased to $135,163,000 as of March 31, 2021, compared to $96,490,000 at the end of 2020, marking an increase of 40.1%[14] - Shareholders' equity decreased to $228,690,000 as of March 31, 2021, down 6.2% from $243,905,000 at the end of 2020[14] - Total contractual obligations as of March 31, 2021, amounted to $144.1 million, including debt obligations and operating lease obligations[165] Clinical Trials and Development - The pivotal HOPE-B trial for the lead gene therapy candidate, Etranacogene dezaparvovec, showed a mean FIX activity increase from ≤ 2% to 37.2% at 26 weeks, meeting the primary endpoint[90] - During the 26-week follow-up, there was an 83% reduction in total bleeding events compared to the observational lead-in phase, with only three spontaneous bleeds requiring treatment[92] - The Phase 1b/II clinical trial for AMT-130 in Europe is expected to begin enrolling patients in the second half of 2021, aiming to establish safety and optimal dosing[99] - The company is responsible for completing the HOPE-B clinical trial and manufacturing process validation for the product under the CSL Behring Agreement[36] - The company plans to incorporate FIX activity and bleeding rates at 52 weeks as additional co-primary endpoints in the HOPE-B study following interactions with the FDA and EMA[92] Regulatory and Collaboration Agreements - The company entered into a collaboration agreement with CSL Behring, receiving an upfront cash payment of $450 million and potential additional payments of up to $1.6 billion based on regulatory and commercial milestones[35] - The company did not recognize any revenue related to the CSL Behring License Revenue due to contingent payments not being fulfilled[38] - The collaboration with Bristol-Myers Squibb (BMS) allows the company to receive up to $217 million in milestone payments for each of the four Collaboration Targets[42] - The company did not record any License Revenue during the three months ended March 31, 2021, due to significant uncertainty surrounding the development of gene-therapy product candidates[46] Risks and Challenges - The ongoing COVID-19 pandemic has materially affected the company's operations, causing disruptions in raw material supply and clinical trial processes[186] - None of the company's product candidates have been approved for commercial sale, and significant revenue generation is not expected before 2022[188] - Clinical trials are subject to delays and may not demonstrate the safety and efficacy of product candidates, impacting future development[191] - The company may need to raise additional funding, which could be difficult to obtain on acceptable terms, impacting product development efforts[182] - The company may face challenges in enrolling eligible patients for clinical trials due to the rarity of the diseases targeted, potentially delaying or abandoning trials[197]