rare genetic diseases companies
Companies whose enriched business profile is tagged “rare genetic diseases”, drawn from their SEC filings + website text. Sorted by market capitalization; every figure is delayed data from SEC EDGAR + Finnhub, shown with its as-of date.
- Alnylam Pharmaceuticals (ALNY) Health Care — A commercial-stage RNAi therapeutics pioneer with six FDA-approved siRNA medicines targeting rare genetic and cardiometabolic diseases. ask →
- Biogen (BIIB) Health Care — Biogen is a global biotechnology company developing and commercializing therapies across neurology, immunology, and rare genetic disease. ask →
- BioMarin Pharmaceutical (BMRN) Health Care — BioMarin is a global rare disease biotech company delivering genetic medicines across enzyme therapies, growth disorders, metabolic diseases, and gene therapies with a proven $3.2 billion commercial franchise. ask →
- Ionis Pharmaceuticals (IONS) Health Care — Pioneer in RNA-targeted antisense medicines with seven commercialized products and expanding late-stage pipeline across rare neurological and cardiometabolic diseases, now executing independent U.S. launches while generating royalty revenue from major pharma partnerships. ask →
- BEAM THERAPEUTICS INC (BEAM) Health Care — Beam Therapeutics is developing base editing medicines—a precision genetic cure approach—for serious inherited diseases like sickle cell disease and rare genetic disorders. ask →
- Ultragenyx (RARE) Health Care — A biopharmaceutical company developing and commercializing novel therapies for rare and ultra-rare genetic diseases with high unmet medical need. ask →
- INTELLIA THERAPEUTICS INC (NTLA) Health Care — CRISPR-based genome editing company developing in vivo therapies for rare genetic diseases including hereditary angioedema and ATTR amyloidosis. ask →
- CAPRICOR THERAPEUTICS INC (CAPR) Health Care — Capricor is a clinical-stage biotech developing cell and exosome therapies for rare genetic disorders, with its lead candidate Deramiocel under FDA review for Duchenne muscular dystrophy treatment. ask →
- DESIGN THERAPEUTICS INC (DSGN) Health Care — Design Therapeutics is a clinical-stage biotech company pioneering GeneTAC® small molecules to treat rare genetic diseases like Friedreich ataxia. ask →
- EDITAS MEDICINE INC (EDIT) Health Care — A pioneering CRISPR gene editing company developing in vivo therapies for genetic diseases, with lead candidate EDIT-401 targeting hyperlipidemia through LDL receptor upregulation. ask →
- ROCKET PHARMACEUTICALS INC (RCKT) Health Care — Rocket Pharmaceuticals develops AAV and lentiviral gene therapies for rare genetic and cardiac diseases with a focus on monogenic disorders. ask →
- VANDA PHARMACEUTICALS INC (VNDA) Health Care — Niche biopharmaceutical company commercializing approved therapies for psychiatry, sleep disorders, and multiple sclerosis while pursuing pipeline expansion in dermatology, rare diseases, and oncology. ask →
- FULCRUM THERAPEUTICS INC (FULC) Health Care — Fulcrum Therapeutics discovers and develops treatments for genetically-defined rare diseases, with pociredir as a lead clinical-stage program. ask →
- CAMP4 THERAPEUTICS CORP (CAMP) Health Care — CAMP4 is pioneering a novel RNA-targeting platform to upregulate deficient genes in rare genetic diseases, starting with SYNGAP1-related developmental epilepsy. ask →
- ARCTURUS THERAPEUTICS HOLDINGS INC (ARCT) Health Care — Arcturus Therapeutics develops self-amplifying mRNA therapeutics and vaccines, with the first approved sa-mRNA COVID vaccine (KOSTAIVE) generating revenue while advancing pipeline programs for rare genetic diseases. ask →
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Data: SEC EDGAR (filings) + Finnhub (fundamentals, delayed daily-close). Figures shown with their as-of date.
Last updated 2026-07-10.
Informational only — NOT financial advice. All figures are delayed daily-close data from SEC EDGAR & Finnhub, shown with their as-of date.