uniQure appoints Kylie O’Keefe as Chief Customer and Strategy Officer

LEXINGTON, Mass. and AMSTERDAM - uniQure N.V. (NASDAQ:QURE), whose stock has surged over 200% in the past year and currently commands a market capitalization of $895 million, has appointed Kylie O’Keefe as Chief Customer and Strategy Officer, effective June 6, 2025, according to a company press release. InvestingPro analysis suggests the stock is currently trading near its Fair Value.

O’Keefe will lead the development and execution of uniQure’s global commercialization strategy for AMT-130, the company’s investigational gene therapy for Huntington’s disease. Her responsibilities will include overseeing all commercial functions and medical affairs as the company prepares for a potential U.S. commercial launch in 2026. The appointment comes at a crucial time as InvestingPro data shows the company is rapidly burning through cash, with analysts forecasting a sales decline in the current year.

Prior to joining uniQure, O’Keefe served as Chief Commercial Officer at PTC Therapeutics, where she led global commercial strategy and operations for rare disease products across more than 50 countries. Her experience includes commercial launches of gene therapies, including Upstaza for AADC deficiency.

"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," said Matt Kapusta, chief executive officer of uniQure.

O’Keefe holds a Bachelor’s degree in Biotechnology Innovation from Queensland University of Technology and a Graduate diploma in Managing Medical Product Innovation from Copenhagen Business School.

AMT-130 is being developed as a potential treatment for Huntington’s disease, with the company pursuing an accelerated approval pathway in the United States.

uniQure is a gene therapy company that previously received approval for a hemophilia B treatment. The company is also advancing gene therapies for conditions including refractory temporal lobe epilepsy, ALS, and Fabry disease. Seven analysts have recently revised their earnings expectations upward, according to InvestingPro, which offers 12 additional exclusive insights about uniQure’s financial health and market position.

In other recent news, uniQure has made significant strides in its gene therapy initiatives, particularly with its lead candidate, AMT-130, aimed at treating Huntington’s disease. The company recently held Type B meetings with the U.S. Food and Drug Administration (FDA), which reaffirmed support for an accelerated approval path for AMT-130. This progress aligns with uniQure’s plans to submit a Biologics License Application (BLA) in the first quarter of 2026, with the potential for approval in the latter half of that year. Analysts from H.C. Wainwright have reiterated their Buy rating and a $70 price target for uniQure, citing AMT-130 as a key value driver for the company. Stifel analysts also maintain a Buy rating, highlighting the company’s regulatory interactions and the anticipated 3-year data for AMT-130 expected in late 2025. Additionally, uniQure’s AMT-260 program for mesial temporal lobe epilepsy (mTLE) has shown promising preliminary results, further supporting the company’s innovative gene therapy approach. The FDA’s Breakthrough Therapy designation for AMT-130 underscores the potential for accelerated development and approval. With a strong financial position and multiple clinical data readouts anticipated in the coming years, uniQure continues to attract attention from investors and analysts alike.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.