PepGen: Clearance by Health Canada of CTA for PGN-EDO51

PepGen has received clearance by Health Canada for the CONNECT1-EDO51 Phase 2 clinical trial of PNG-EDO51, an exon 51 skipping therapy.

The first-ever FDA Advisory Committee meeting for a gene therapy intended to treat Duchenne muscular dystrophy received a positive vote for accelerated approval of SRP-9001.

Santhera has shared it’s launch plans for vamorolone in the US, EU, and UK, as well as an update on the FDA mid-cycle review.

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.