gene therapy companies

Companies whose enriched business profile is tagged “gene therapy”, drawn from their SEC filings + website text. Sorted by market capitalization; every figure is delayed data from SEC EDGAR + market data, shown with its as-of date.

• KRYSTAL BIOTECH INC (KRYS) Health Care — Krystal Biotech is a commercial-stage gene therapy company using its proprietary STAR-D platform to treat rare diseases with targeted, redosable genetic medicines. ask →
• PTC THERAPEUTICS INC (PTCT) Health Care — A commercial-stage biopharmaceutical company dedicated to discovering and developing innovative therapies for rare genetic diseases. ask →
• PRECIGEN INC (PGEN) Health Care — A commercial-stage precision medicine company leveraging proprietary adenoviral and CAR-T platforms to address intractable diseases, notably including the FDA-approved RRP therapy Papzimeos. ask →
• Sarepta Therapeutics (SRPT) Health Care — Sarepta Therapeutics is a leader in genetic medicine, specializing in innovative RNA-targeted and gene therapies to treat rare neuromuscular diseases like Duchenne muscular dystrophy. ask →
• TAYSHA GENE THERAPIES INC (TSHA) Health Care — Taysha Gene Therapies is a clinical-stage biotechnology company developing AAV-based gene therapies for rare, monogenic central nervous system disorders. ask →
• MEIRAGTX HOLDINGS PLC (MGTX) Health Care — MeiraGTx is a vertically integrated genetic medicine company developing a pipeline of gene therapies and a unique riboswitch platform for dose-responsive, oral-small-molecule control of gene expression. ask →
• SOLID BIOSCIENCES INC (SLDB) Health Care — A clinical-stage biotechnology company developing gene therapies to treat rare, life-threatening neuromuscular and cardiac diseases. ask →
• DESIGN THERAPEUTICS INC (DSGN) Health Care — Design Therapeutics is a biopharmaceutical company pioneering GeneTAC small-molecule therapies to treat the root causes of serious genetic diseases. ask →
• Fractyl Health, Inc. (GUTS) Health Care — Fractyl Health is a metabolic therapeutics company pioneering procedural and gene therapy treatments for obesity and type 2 diabetes, including its lead asset Revita for post-GLP-1 weight maintenance. ask →
• 4D MOLECULAR THERAPEUTICS INC (FDMT) Health Care — 4D Molecular Therapeutics is a clinical-stage biotechnology company pioneering targeted genetic medicines to transform the treatment of debilitating ophthalmological and pulmonary diseases. ask →
• REGENXBIO INC (RGNX) Health Care — A clinical-stage gene therapy developer leveraging its proprietary AAV platform to create potentially curative, one-time treatments for serious genetic diseases. ask →
• NEUROGENE INC (NGNE) Health Care — Neurogene is a clinical-stage gene therapy company developing a proprietary transgene regulation platform to treat rare, severe neurological disorders like Rett syndrome. ask →
• XOMA ROYALTY CORP (XOMA) Health Care — A lean-infrastructure royalty aggregator that generates non-dilutive capital for biotech developers in exchange for long-duration milestone and royalty rights across a diversified 120+ asset portfolio spanning oncology, rare disease, and specialty therapeutics. ask →
• OPUS GENETICS INC (IRD) Health Care — Opus Genetics is a clinical-stage gene therapy company developing a portfolio of durable, one-time treatments for rare inherited retinal diseases. ask →
• OCUGEN INC (OCGN) Health Care — Ocugen is a clinical-stage biotechnology company developing gene-agnostic modifier gene therapies to address significant unmet medical needs in blindness and retinal diseases. ask →
• BENITEC BIOPHARMA INC (BNTC) Health Care — Benitec Biopharma is a biotechnology company advancing a proprietary 'silence and replace' gene therapy platform to address rare genetic disorders like OPMD. ask →
• ROCKET PHARMACEUTICALS INC (RCKT) Health Care — Rocket Pharmaceuticals is a commercial-stage biotech company developing AAV-based gene therapies for rare cardiovascular conditions and genetic disorders. ask →
• ABEONA THERAPEUTICS INC (ABEO) Health Care — Abeona Therapeutics is a commercial-stage biopharmaceutical firm providing the first autologous cell-based gene therapy for the treatment of rare, debilitating RDEB skin wounds. ask →
• LEXEO THERAPEUTICS INC (LXEO) Health Care — Lexeo Therapeutics is a clinical-stage genetic medicine company developing pioneering gene therapies for cardiovascular diseases with high unmet needs. ask →
• VOYAGER THERAPEUTICS INC (VYGR) Health Care — Voyager Therapeutics is a gene therapy company utilizing its proprietary TRACER AAV platform to develop intravenous, brain-penetrant treatments for severe neurological diseases. ask →
• Tenaya Therapeutics, Inc. (TNYA) Health Care — Tenaya Therapeutics is a clinical-stage precision medicine company developing curative, genetically-driven therapies to treat the root causes of heart disease. ask →
• MaxCyte, Inc. (MXCT) Health Care — MaxCyte provides high-performance, non-viral cell engineering platforms and gene-editing assessment services that enable the development of next-generation cell and gene therapies. ask →

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Data: SEC EDGAR (filings) + market data (fundamentals, delayed daily-close). Figures shown with their as-of date.

Last updated 2026-10-08.

Informational only — NOT financial advice. All figures are delayed daily-close data from SEC EDGAR & market data, shown with their as-of date.