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 ...

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 ...