~ Proven biotech executive to lead commercialization of AMT-130 in Huntington’s disease ~

LEXINGTON, Mass. and AMSTERDAM, June 11, 2025 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced the appointment of Kylie O’Keefe as Chief Customer and Strategy Officer, effective June 6, 2025. In this role, Ms. O’Keefe will lead the development and execution of uniQure’s global commercialization strategy for AMT-130, the Company’s investigational gene therapy for the treatment of Huntington’s disease. Her responsibilities include all commercial functions and medical affairs. Ms. O’Keefe was most recently Chief Commercial Officer at PTC Therapeutics.

“We are very pleased to welcome Kylie to the uniQure executive leadership team as we plan for the potential U.S. commercial launch of AMT-130 in 2026,” said Matt Kapusta, chief executive officer of uniQure. “Kylie brings deep commercial expertise in rare diseases, and her knowledge of Huntington’s disease and execution of gene therapy launches will be invaluable as we prepare to transition to a commercial-stage biotechnology company.”

Ms. O’Keefe is an accomplished business leader with broad biopharmaceutical experience and a proven track record in rare diseases and gene therapy. Most recently, she served as Chief Commercial Officer at PTC Therapeutics, where she led global commercial strategy, operations and portfolio management for multiple rare neurology and metabolic commercial products across more than 50 countries. During her tenure, Ms. O’Keefe led several strategically significant commercial launches and supported corporate strategy and pipeline development for both small molecules and gene therapies including Upstaza™ (AADC deficiency). She also directed the development and execution of reimbursement strategies, including payer engagement and health economic assessments, and led business development and investor relations. Earlier in her career, Ms. O’Keefe held key roles at LEO Pharma where she launched a portfolio of pharmaceutical products in more than 30 countries. Ms. O’Keefe holds a Bachelor’s degree in Biotechnology Innovation from Queensland University of Technology in Brisbane, Australia and a Graduate diploma in Managing Medical Product Innovation from Copenhagen Business School in Frederiksberg, Denmark.

“I am thrilled to join uniQure at such an exciting time for the company,” commented Ms. O’Keefe. “AMT-130 has the potential to be the first disease-modifying treatment for Huntington’s disease. With a clear and aligned path towards accelerated approval in the U.S., uniQure is well positioned to deliver a potentially historic breakthrough in Huntington’s disease. I look forward to leveraging my experiences in rare disease and gene therapy, and to working with the talented team at uniQure to advance its mission of addressing the urgent needs of Huntington’s patients.”

About uniQure

uniQure is delivering on the promise of gene therapy – single treatments with potentially curative results. The approvals of uniQure’s gene therapy for hemophilia B – an historic achievement based on more than a decade of research and clinical development – represent a major milestone in the field of genomic medicine and ushers in a new treatment approach for patients living with hemophilia. uniQure is now advancing a pipeline of proprietary gene therapies for the treatment of patients with Huntington's disease, refractory temporal lobe epilepsy, ALS, Fabry disease, and other severe diseases. www.uniQure.com

uniQure Forward-Looking Statements

This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as "anticipate," "believe," "could," “establish,” "estimate," "expect," "goal," "intend," "look forward to", "may," "plan," "potential," "predict," "project," “seek,” "should," "will," "would" and similar expressions. Forward-looking statements are based on management's beliefs and assumptions and on information available to management only as of the date of this press release. Examples of these forward-looking statements include, but are not limited to, statements concerning the Company’s commercialization plans; the Company’s ability to deliver potentially life-changing therapy to people living with Huntington’s disease and related timeline for doing so; the potential clinical and functional effects of AMT-130; and the Company’s plans to continue clinical development of AMT-130. The Company’s actual results could differ materially from those anticipated in these forward-looking statements for many reasons. These risks and uncertainties include, among others: risks associated with the Company’s Phase I/ll clinical trials of AMT-130, including the risk that interim data from the trials may not be predictive of later data readouts that will serve as a basis for further regulatory interactions, may not support a Biologics License Application (BLA) submissions or accelerated approvals, may not be satisfactory to the FDA and other regulators, and new analyses of existing data and results may produce different conclusions than established as of the date hereof; risks related to the Company’s current and future interactions with regulatory authorities, which may affect the initiation, timing and progress of clinical trials, its BLA submission plans and pathways to regulatory approval; risks related to the Company’s ability to pursue business development efforts with respect to AMT-130; uncertainties as to the FDA’s and other regulatory authorities’ interpretation of the data from the Company’s Phase I/ll clinical trials of AMT-130 and acceptance of the Company’s clinical programs and the regulatory approval process; later developments with the FDA and other regulators that could be inconsistent with the feedback received to date; the Company’s ability to continue to build and maintain the Company infrastructure and personnel needed to achieve its goals; the Company’s effectiveness in managing current and future clinical trials and regulatory processes; the continued development and acceptance of gene therapies; the Company’s ability to demonstrate the therapeutic benefits of its gene therapy candidates in clinical trials; the Company’s ability to obtain, maintain and protect intellectual property; and the Company’s ability to fund its operations and to raise additional capital as needed. These risks and uncertainties are more fully described under the heading "Risk Factors" in the Company’s periodic filings with the U.S. Securities & Exchange Commission (“SEC”), including its Annual Report on Form 10-K filed with the SEC on February 27, 2025 and in other filings that the Company makes with the SEC from time to time. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and the Company assumes no obligation to update these forward-looking statements, even if new information becomes available in the future.

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