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.

Senti Biosciences Holdings, Inc. is a clinical-stage synthetic biology company developing next-generation cell and gene therapies using its proprietary Gene Circuit platform and Regulator Dial technology platform. The company's lead clinical asset, SENTI-202, was a logic-gated CAR-NK cell therapy for relapsed/refractory AML that received RMAT designation from FDA. On July 15, 2026 the company announced a strategic transaction spinning off its Gene-Circuit-enabled pipeline (including SENTI-202) into a newly formed private company controlled by Celadon Partners (Senti's largest investor) in exchange for a contingent value right worth up to $60M in milestone payments over seven years. Post-transaction, the retained company plans to focus on early-stage Regulator Dial programs including a controllable gene therapy for Rett Syndrome and armored tumor-infiltrating lymphocytes (TILs) for solid tumors.