uniQure N.V. has appointed Kylie O'Keefe as Chief Customer and Strategy Officer, effective June 6, 2025, to spearhead the global commercialization strategy for AMT-130, the company's investigational gene therapy for Huntington's disease. The appointment signals uniQure's preparation for a potential U.S. commercial launch of AMT-130 in 2026.
Strategic Leadership for Gene Therapy Launch
O'Keefe will oversee all commercial functions and medical affairs as uniQure transitions toward becoming a commercial-stage biotechnology company. "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."
The new executive brings extensive biopharmaceutical experience, most recently serving 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 at PTC Therapeutics, O'Keefe directed several strategically significant commercial launches and supported corporate strategy and pipeline development for both small molecules and gene therapies, including Upstaza™ for AADC deficiency.
AMT-130's Therapeutic Potential
AMT-130 represents a potentially groundbreaking advancement in Huntington's disease treatment. "AMT-130 has the potential to be the first disease-modifying treatment for Huntington's disease," commented O'Keefe. "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."
The investigational gene therapy is currently undergoing Phase I/II clinical trials. According to uniQure's forward-looking statements, the company faces various risks associated with these trials, including the possibility that interim data may not be predictive of later readouts or may not support Biologics License Application submissions or accelerated approvals.
Commercial Expertise and Background
O'Keefe's commercial expertise extends beyond PTC Therapeutics, having previously held key roles at LEO Pharma where she launched a portfolio of pharmaceutical products in more than 30 countries. Her experience includes developing and executing reimbursement strategies, payer engagement, health economic assessments, and leading business development and investor relations.
She 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.
uniQure's Gene Therapy Pipeline
uniQure has established itself in the gene therapy field with approved treatments for hemophilia B, representing what the company describes as "an historic achievement based on more than a decade of research and clinical development." Beyond AMT-130, the company is advancing a pipeline of proprietary gene therapies for patients with refractory temporal lobe epilepsy, ALS, Fabry disease, and other severe diseases.
The company's approach focuses on delivering "single treatments with potentially curative results," positioning gene therapy as a transformative treatment modality for patients with severe medical needs.
