ARTICLE | Product Development
Two gene therapies for Duchenne muscular dystrophy advance to Phase III as a preclinical program attracts pharma acquisition
By Lauren Martz, Executive Director, Biopharma Intelligence
November 25, 2024 11:16 PM UTC
Three announcements last week signal that innovation is still alive and advancing in Duchenne muscular dystrophy gene therapy despite the approval of Elevidys. They also provide the latest example of FDA’s willingness to embrace the accelerated approval pathway for gene therapies.
Last Monday, RegenxBio Inc. (NASDAQ:RGNX) and Genethon S.A. each announced plans to advance gene therapies into pivotal DMD trials next year, while Novartis AG (SIX:NOVN; NYSE:NVS) announced it was acquiring preclinical-stage Kate Therapeutics Inc. (NASDAQ:KATE)…
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- Source: https://www.biocentury.com/article/654297/elevidys-isn-t-slowing-innovation-in-dmd-gene-therapy