Duchenne Muscular Dystrophy

Duchenne muscular dystrophy (DMD), which occurs in approximately 1 in 3500-5000 live male births, causes a lack of functional dystrophin that leads to stress and tears of muscle cell membranes, resulting in muscle cell death. The dystrophin protein maintains the integrity of muscle fibers and acts as a shock absorber through its role as the foundation of a group of proteins that connects the inner and outer elements of muscle cells. DMD patients suffer from progressive muscle weakness that typically starts in boys at a very young age. Those living with the condition often require special aid and assistance throughout their lives and have significantly shortened life expectancy.

We are developing AOCs to treat the underlying cause of DMD. The oligonucleotides in our AOCs are designed to promote the skipping of specific exons to allow the production of the dystrophin gene product in patients with DMD. In preclinical studies, we observed that treatment of an mdx mouse, a widely used animal model for DMD, with an AOC caused a greater than 50-fold increase in exon skipping compared to an equimolar dose of the unconjugated oligonucleotide.

DMD Exons Amenable to Skipping:

Several DMD exons are amenable to skipping. Avidity is pursing AOCs for Exons 51, 45 and 44, representing ~30% of mutations amenable to skipping.

Our initial development efforts in DMD are focused on AOCs that can induce exon skipping specifically for Exons 44, 51 and 45. We have received IND clearance from the U.S. Food and Drug Administration (FDA) to proceed with a Phase 1/2 study of AOC 1044 for people with Duchenne muscular dystrophy (DMD) who have mutations amenable to exon 44 skipping. AOC 1044 is now advancing into the clinic. 

DMD Webinar

In September 2021, our CSO, Art Levin, participated in a DMD panel where he described our AOC approach to DMD. To view the panel, please click on the webinar link below.

Duchenne Muscular Dystrophy: Charting the Path for New Therapeutics and Better Care

Webinar Speaker Collaborations Co-hosted by WuXi AppTec, Parent Project Muscular Dystrophy & CureDuchenne

New Targets. New Modalities. New Directions : Panel Participation by Art Levin, Chief Scientific Officer – September 9, 2021

Dr. Levin’s panel begins at ~ the 1:40 minute mark