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Matthew Klein

Chief Executive Officer, Ptc Therapeutics

Search every verified Matthew Klein interview, podcast appearance, and on-the-record quote — each transcript cross-checked by AI and human review to confirm speaker identity. Matthew Klein, Chief Executive Officer of PTC Therapeutics, has been discussing the company's progress in developing treatments for rare diseases, including its move toward profitability and self-sufficiency. In a May 2025 podcast, he described PTC's history as a pioneer in RNA-targeted therapies and highlighted a partnership with Novartis for the Huntington's disease program PTC518, which he said included $1 billion upfront and $1.9 billion in potential milestones. He stated that the company closed the first quarter of 2025 with over $2 billion in cash and emphasized a philosophy of focusing on areas where the company can differentiate itself. Klein has also provided updates on specific pipeline programs. In January 2025, he participated in a webinar hosted by the Friedreich's Ataxia Research Alliance regarding the company's submission of a New Drug Application to the U.S. Food and Drug Administration for vatiquinone, a candidate for Friedreich's ataxia, noting that PTC had requested approval for patients aged six and above. In earlier appearances, he discussed the company's gene therapy platform, including the approved therapy Upstaza for AADC deficiency, and its strategy of targeted delivery to specific anatomical locations. He also spoke about clinical trial adaptations during the COVID-19 pandemic, including remote visits and expanded site access for a mitochondrial disease study.

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