Mark Stahl, MD, PhD Archives

2021 FUTURES: Exon Skipping Panel

Information and updates on exon skipping therapies and research from experts in the Duchenne community.

Ash Dugar, PhD

Senior Vice President, Medical Affairs

Dyne Therapeutics

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Kevin Eggan, PhD

Group Vice President, Head of Research and Early Development

BioMarin

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Leslie Magnus, MD

Vice President, Medical Affairs

NS Pharma

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James McArthur, PhD

President and Chief Executive Officer

PepGen

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Lianna Orlando, PhD

Senior Director of Research

CureDuchenne

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Mark Stahl, MD, PhD

Senior Medical Director, Clinical Development

Avidity Biosciences

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Deanna Tucker, PharmD

Senior Medical Science Liaison

Sarepta Therapeutics

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Avidity Biosiences

Advancing Exon Skipping AOCs for the Treatment of Duchenne Muscular Dystrophy

Avidity Biosciences is a biotechnology company based in La Jolla, California. At Avidity, we are driven by our mission to improve the lives of people affected by diseases with limited therapeutic options, such as Duchenne muscular dystrophy (DMD). We are doing this by realizing the broad and disruptive potential of our Antibody Oligonucleotide Conjugates (AOC™) platform. AOCs combine the specificity of monoclonal antibodies and the precision of oligonucleotides. Avidity is advancing three different exon skipping AOC drugs designed to treat the root cause of DMD in individuals who are amenable to skipping exons 44, 45 or 51. Our first program in development for the treatment of DMD is called AOC 1044 and is designed to treat individuals who are amenable to exon 44 skipping treatments. We are planning to initiate our first clinical trial with AOC 1044 by the end of 2022.

Mark Stahl, MD, PhD

Senior Medical Director, Clinical Development

Avidity Biosciences

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Exon Skipping and Read Through Agents Panel

A brief introduction on the rationale behind mechanisms of endogenous dystrophin restoration, like exon skipping and read through agent therapies, followed by critical information and updates from the companies in this space. Moderated discussion included.