Avidity Biosciences: Granted FDA Fast Track Designation for AOC 1044

Avidity Biosciences has been granted Fast Track designation to AOC 1044 for the treatment of Duchenne muscular dystrophy (DMD) in people with mutations amenable to exon 44 skipping (DMD44) by the FDA in the United States.

NS Pharma has received FDA clearance to initiate a Phase II Study for NS-089/NCNP-02, an exon 44 skipping candidate for Duchenne muscular dystrophy.

REGENXBIO has received FDA fast track designation for RGX-202, their gene therapy candidate for Duchenne muscular dystrophy.

Edgewise Therapeutics has published a new article in the Journal of Clinical Investigation discussing EDG-5006, their oral small-molecule muscle stabilizer for Duchenne and Becker muscular dystrophy.

Capricor Therapeutics has presented the 18-month results from its HOPE-2 open-label extension (OLE) study with lead asset, CAP-1002, for the treatment of Duchenne muscular dystrophy.

PepGen has presented clinical and nonclinical data at the 2023 Annual Muscular Dystrophy Association (MDA) Clinical and Scientific Conference, including an update on a Canadian clinical trial for Duchenne.

Dyne Therapeutics has received FDA Orphan Drug and Rare Pediatric Designations for DYNE-251 for the treatment of Duchenne muscular dystrophy.

Solid Biosciences have provided a community update that highlights activities from 2022 and details on the IGNITE DMD Phase 1 and 2 Clinical Trial.

Wave Life Sciences plans to initiate Part B of WVE-N531, their open-label study for those with Duchenne muscular dystrophy amendable to exon 53 skipping.

Avidity Biosciences has provided an update on the development of AOC 1044 – an Antibody Oligonucleotide Conjugates (AOCs™) designed to use an antibody to deliver an exon 44-skipping oligonucleotide to muscle and heart cells for individuals with Duchenne.