Companies in #Gene Therapy

CureDuchenne is a nonprofit focused on accelerating Duchenne muscular dystrophy research and care. Founded in 2003, it has funded multiple DMD gene therapy and exon-skipping programs and is a major patient-advocacy partner in the DMD therapeutics space.

Grace Science is a clinical-stage biotechnology company developing gene therapies for ultra-rare neurological diseases, including NGLY1 Deficiency and GM3 Synthase Deficiency. The company is led by founder and CEO Ken Drazan and is built around a parent-driven drug development model, with its lead gene therapy program targeting a disease that affects the founder's own daughter and a small global patient population.

London-based gene therapy company developing treatments for Parkinson's disease, radiation-induced xerostomia, and inherited retinal diseases, using proprietary Riboswitch gene regulation platform.

Sarepta Therapeutics is a commercial-stage biopharmaceutical company focused on precision genetic medicines for rare neuromuscular and central nervous system diseases.