~ Genezen to manufacture global commercial supply of HEMGENIX® and support uniQure's pipeline programs as a preferred customer ~ Genezen agreed to acquire uniQure's commercial-scale gene-therapy manufacturing facility for total consideration of $25 million, comprised of $12.5 million of newly issued Series C Preferred Stock and a $12.5 million convertible note. In addition, Matt Kapusta, chief executive officer of uniQure, will join the Board of Genezen upon the closing of the transaction, which is expected ...

~ Designation based on 24-month interim Phase I/II clinical data for AMT-130 announced in December 2023 ~ LEXINGTON, Mass. and AMSTERDAM, June 03, 2024 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation for its investigational gene therapy AMT-130 for the treatment of Hunti ...

uniQure Announces First Quarter 2024 Financial Results and Highlights Recent Company Progress Exhibit 99.1 ~ On track to initiate FDA interaction regarding AMT-130 in second quarter of 2024 and provide a clinical update from the Phase I/II trials in mid-2024 ~ ~ Clinical trial initiation for Fabry disease on track to begin in second quarter of 2024, followed by refractory mesial temporal lobe epilepsy and SOD1-ALS in third quarter of 2024 ~ ~ Comprehensive review of operations and options to reduce expenses ...

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 March 31, 2024 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 (Sta ...

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 ...

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington D.C. 20549 The Netherlands 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, 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) ...

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 ...