The definitive directory of gene therapy, cell therapy, and gene editing companies developing curative genetic medicines for rare disease, oncology, and beyond. Curated for pharma BD teams, investors tracking the CGT pipeline, and operators sourcing gene and cell therapy partners.
First company to commercialize CRISPR-based gene editing with Casgevy (Vertex partnership) for sickle cell disease and beta-thalassemia. Expanding into in vivo editing and CAR-T for oncology.
Leader in in vivo CRISPR gene editing. Lead program NTLA-2001 targets transthyretin amyloidosis with groundbreaking clinical data. Broadening into hereditary angioedema and oncology.
Pioneer in CAR-T therapy with Yescarta and Tecartus. Investing $1.6B+ in next-gen in vivo cell therapy through Pregene Biopharma acquisition. Leading CAR-T manufacturer globally.
Pioneering base editing technology that rewrites single DNA letters without double-strand breaks. Clinical programs in sickle cell disease, AATD, and GSD1a. Pfizer collaboration for in vivo programs.
Leading gene therapy company in neuromuscular disorders. Elevidys approved for Duchenne muscular dystrophy. Multiple AAV-based therapies in development with focus on safety optimization.
Commercial-stage gene therapy company (formerly Bluebird Bio). FDA-approved Zynteglo, Skysona, and Lyfgenia for sickle cell, beta-thalassemia, and cerebral adrenoleukodystrophy.
FDA-approved Vyjuvek gene therapy for dystrophic epidermolysis bullosa using proprietary STAR-D skin delivery platform. Expanding into NSCLC with inhaled gene therapy.
Developer of Carvykti (ciltacabtagene autoleucel) CAR-T therapy for multiple myeloma in partnership with J&J/Janssen. One of the fastest-growing CAR-T franchises globally.
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The BioMed Nexus Gene and Cell Therapy Directory is the most comprehensive listing of companies developing genetic medicines across gene editing, CAR-T, AAV gene therapy, in vivo delivery, and cell-based therapeutics. Updated regularly by our editorial team, this directory covers CRISPR and base editing companies, CAR-T and immune cell therapy developers, AAV vector companies, in vivo gene delivery platforms, rare disease gene therapy specialists, CGT manufacturing platforms, and autoimmune cell therapy pioneers.
Whether you are a pharma BD team evaluating gene therapy licensing opportunities, an investor tracking the CGT pipeline, or a clinical operations team sourcing cell therapy manufacturing, this directory provides the structured data and organization profiles you need to make informed decisions.
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