rare disease therapeutics companies

Companies whose enriched business profile is tagged “rare disease therapeutics”, 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.

• ASTRAZENECA PLC (AZN) Health Care — AstraZeneca is a global, science-led biopharmaceutical company focused on transforming patient outcomes in oncology, rare diseases, and chronic conditions through a robust and diverse R&D pipeline. ask →
• Alnylam Pharmaceuticals (ALNY) Health Care — Alnylam is a pioneer in RNA interference (RNAi) therapeutics, using gene-silencing technology to treat rare genetic diseases and cardiovascular conditions. ask →
• BridgeBio Pharma, Inc. (BBIO) Health Care — BridgeBio is a portfolio-based biotech firm focused on translating genetic science into precision therapies for patients with severe genetic disorders. ask →
• RHYTHM PHARMACEUTICALS INC (RYTM) Health Care — Rhythm Pharmaceuticals is a biopharmaceutical company specializing in precision therapies for rare genetic and neuroendocrine disorders characterized by severe obesity and hyperphagia. ask →
• CATALYST PHARMACEUTICALS INC (CPRX) Health Care — Catalyst Pharma commercializes rare disease therapies with a focus on neuromuscular and neuromuscular-inflammation disorders, generating strong cash flow from established and newly approved products. ask →
• DYNE THERAPEUTICS INC (DYN) Health Care — Dyne Therapeutics is a clinical-stage biotechnology company developing targeted muscle therapeutics for rare diseases using its proprietary FORCE platform. ask →
• VERA THERAPEUTICS INC CLASS A (VERA) Health Care — Vera Therapeutics is a commercial-stage biotech pioneer targeting B-cell-driven autoimmune diseases, currently focused on its lead BAFF/APRIL inhibitor for IgA nephropathy. ask →
• ANI PHARMACEUTICALS INC (ANIP) Health Care — ANI Pharmaceuticals is a specialized biopharma company focused on acquiring, developing, and marketing both branded and generic prescription drugs. ask →
• Hemab Therapeutics Holdings, Inc. (COAG) Health Care — Hemab Therapeutics is a clinical-stage biotechnology company developing innovative bispecific and monovalent antibody therapies for rare blood coagulation disorders. ask →
• WAVE LIFE SCIENCES LTD (WVE) Health Care — Wave Life Sciences is a clinical-stage biotech company developing stereopure nucleic acid therapeutics via its proprietary PRISM platform to treat genetically defined diseases. ask →
• REZOLUTE INC (RZLT) Health Care — Rezolute is a late-stage biotech developer of ersodetug, an insulin receptor-targeting antibody aimed at treating rare forms of refractory hypoglycemia. ask →
• LARIMAR THERAPEUTICS INC (LRMR) Health Care — Larimar Therapeutics is a biotechnology company developing protein replacement therapies, primarily focused on nomlabofusp for the treatment of Friedreich's ataxia. ask →
• NIAGEN BIOSCIENCE INC (NAGE) Consumer Staples — Niagen Bioscience is an anti-aging bioscience company advancing NAD+ science through its proprietary Niagen ingredient used in supplements, compounded medicines, and clinical research. 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.