Companies in #Gene Therapy

Clinical-stage gene therapy company developing treatments to reverse or prevent blindness, including programs for X-linked retinoschisis, GUCY2D-associated blindness and MYO7A-associated Usher syndrome.

Ophthalmic gene therapy company founded to restore and improve vision in patients with rare retinal diseases; its XLRP gene therapy improved visual acuity in a pivotal trial, setting up a regulatory filing.

Global contract development and manufacturing organization (CDMO) providing drug delivery technologies, development, manufacturing, biologics, gene therapy and consumer health services; a privately held subsidiary of Novo Holdings.

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.

Epigenetic therapies for facioscapulohumeral muscular dystrophy (FSHD). Lead program EPI-321 targets the genetic root cause. Also researching alpha-1 antitrypsin deficiency and familial hypercholesterolemia.

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.

New York City-based clinical-stage genetic medicine company advancing disease-modifying gene therapies for genetic cardiovascular conditions and genetic conditions of the central nervous system; in September 2026 it agreed to acquire Mantle Therapeutics to add Friedreich ataxia assets.

Clinical-stage biotechnology company developing precision gene therapies for rare genetic neurodevelopmental disorders.

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

Passage Bio is a clinical-stage genetic medicines company developing gene therapies for central nervous system diseases, built around a research collaboration with the Gene Therapy Program at the University of Pennsylvania's Perelman School of Medicine. Its lead asset, PBFT02, is an AAV1 gene therapy delivering a functional granulin (GRN) gene for the treatment of frontotemporal dementia caused by progranulin deficiency, and is the sole program the company continues to advance after electing to wind down its earlier GM1 gangliosidosis, Krabbe disease and metachromatic leukodystrophy candidates. In June 2026 Passage Bio agreed an all-stock merger with Remix Therapeutics, a clinical-stage company developing small-molecule modulators of RNA processing; on completion the combined company will be renamed Remix Therapeutics, trade on Nasdaq as RMTX, and pursue Remix's lead mRNA degrader REM-422 in adenoid cystic carcinoma and AML/HR-MDS, with legacy Passage Bio holders retaining a minority of the combined entity. The company has flagged going-concern doubt on a declining cash balance and an accumulated deficit of roughly $720 million, and the transaction is subject to a stockholder vote including a proposed reverse stock split.

Commercial-stage biopharmaceutical company developing gene and cell therapies for difficult-to-treat diseases, with a focus on immuno-oncology and precision medicine; its AdenoVerse immunotherapeutic platform received FDA platform technology designation in September 2026.

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.