Capricor Therapeutics: Positive 18-Month Results from HOPE-2 Open-Label Extension Study of CAP-1002

Capricor Therapeutics has shared positive 18-month results from its ongoing HOPE-2 open label extension (OLE) study of CAP-1002 in patients with later-stage Duchenne muscular dystrophy.

REGENXBIO has initiated Phase I/II of AFFINITY DUCHENNE™, a gene therapy clinical trial of RGX-202 in the United States.

PepGen has provided an end of year update for the Duchenne muscular dystrophy community. Please see their newsletter from Q4 2022.

Santhera Pharmaceuticals and ReveraGen BioPharma, Inc have announced that the U.S. Food and Drug Administration (FDA) has
accepted the new drug application (NDA) for vamorolone for the treatment of Duchenne muscular
dystrophy.

Sarepta Therapeutics has submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for the accelerated approval of SRP-9001, an investigational gene therapy for Duchenne muscular dystrophy.

PepGen has announced the results from their completed Phase 1 healthy normal volunteer (HNV) trial of PGN-EDO51, the company’s lead exon skipping product for Duchenne muscular dystrophy.

Roche is now recruiting caregivers and patients who are able to offer their perspectives on completing the North Star Ambulatory Assessment (NSAA) or Performance of Upper Limb (PUL) as part of a new research study.

Edgewise Therapeutics has announced positive 4-month interim results from the ARCH open label study of EDG-5506 in adults with Becker muscular dystrophy (BMD).

The U.S. Food and Drug Administration (FDA) has authorized a clinical trial of EDG-5506, an investigational orally administered small molecule myosin modulator designed to protect injury-susceptible fast skeletal muscle fibers in Duchenne and Becker muscular dystrophy.