Companies in #Duchenne Muscular Dystrophy (DMD)
Found 3 companies operating in this sector.
REGENXBIO is a clinical-stage biotechnology company developing gene therapies using its proprietary AAV (adeno-associated virus) NAV technology platform. The company is advancing a pipeline of one-time gene therapies for rare and serious diseases including Duchenne muscular dystrophy (RGX-202), Hunter syndrome (RGX-121), and wet age-related macular degeneration (RGX-314, partnered with AbbVie).
Vertex Pharmaceuticals is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. Vertex is the leading developer of cystic fibrosis therapies (Trikafta/Kaftrio franchise), brought the first FDA-approved CRISPR-based therapy to market (Casgevy, with CRISPR Therapeutics), and has a late-stage pipeline in acute pain, sickle cell disease, beta thalassemia, and Duchenne muscular dystrophy.
Wave Life Sciences is a clinical-stage RNA medicines company using its proprietary PRISM platform to design stereopure oligonucleotides that target genetic diseases, metabolic disorders, and neurological conditions. Lead programs include WVE-006 for Alpha-1 Antitrypsin Deficiency (AATD), WVE-003 for Huntington's disease, WVE-007 for obesity, and WVE-N531 for Duchenne muscular dystrophy, with a strategic collaboration with GSK to accelerate development of RNA-based therapeutics.