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.
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.
Edgewise Therapeutics is a clinical-stage biopharmaceutical company focused on creating novel precision medicines for the treatment of rare muscle disorders. Our intimate knowledge of integrated muscle physiology at a whole-body level allows us to develop innovative solutions for patients with muscle disease where significant unmet medical need exists. By protecting and improving muscle health, our goal is to dramatically enhance the lives of people living with progressive muscle disorders.
FibroGen accelerates the delivery of innovative treatments that operate at the cutting edge of cancer research. Our teams have identified and are developing potential first-in-class medicines for the treatment of life-threatening conditions such as metastatic pancreatic cancer, locally advanced pancreatic cancer (LAPC), and chemotherapy-induced anemia (CIA). We are keenly focused on leveraging the body’s natural pathways to treat cancer and its related conditions.
Bassem Elmankabadi, MD, ABWS
Executive Medical Director, Clinical Development (Fibrosis/Oncology)
Join us for this pre-recorded presentation and Q&A discussing to learn about a new FDA-approved treatment for Duchenne muscular dystrophy. During the session, Catalyst Pharmaceuticals will provide an overview of how this novel corticosteroid works and its efficacy in Duchenne. You will also learn about the journey to comprehensive support and access through the well-established Catalyst Pathways patient support program.
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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