Industry Presentations

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The Industry Presentations category provides information and updates from biotech and pharma companies with programs focused on Duchenne. These presentations are updated as new information is provided to CureDuchenne.

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


RGX-202:  REGENXBIO’s investigational gene therapy for the treatment of Duchenne Muscular Dystrophy presented by:  Johannaz (Naz) Dastgir, DO, Senior Director, Clinical Development Lead at REGENXBIO

A program update on RGX-202, REGENXBIO’s investigational gene therapy program for the treatment of Duchenne.

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Johannaz Dastgir, DO

Senior Director, Clinical Development Lead