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How a Gene Therapy for Duchenne Muscular Dystrophy Came to Be

  • Broadcast in Science
Pharma Talk Radio

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This podcast dives into the history of creating a gene therapeutic for Duchenne Muscular Dystrophy. Dr Richard Jude Samulski has been studying gene therapy and the use of the AAV vector for decades. Recently, preliminary results from Pfizer were published on a small gene therapy trial for Duchenne Muscular Dystrophy with promising results, and Pfizer is scheduling a larger trial for later this year.

That gene therapy originated from Dr Samulski work and from AskBio, and uses an adeno-associated virus to carry a healthy copy of the dystrophin gene; the virus was injected into boys with DMD, broke into their muscle cells, and replaced their non-working gene.

Dr Samulski sat down with PharmaTalk's Danny McCarthy to discuss the process of creating an AAV vector to carry the gene therapeutic, the mechanisms of the therapy in DMD and what the future of the gene therapy landscape might look like. 


R. Jude Samulski, PhD, is President, Chief Scientific Officer and Co-Founder of Asklepios BioPharmaceutical and Professor of Pharmacology in the UNC School of Medicine. Dr Samulski has worked with AAV for 40 years, and for 25 years, he was director of the University of North Carolina Gene Therapy Center.

Asklepios Bio's Chief Medical Officer, Martin Childers, DO, PhD, will be speaking at the Chief Medical Officer 360˚ summit, October 26-27, 2020, virtually.  

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