Rare diseases companies

Companies whose enriched business profile is tagged “Rare 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.

• ASTRAZENECA PLC (AZN) — Leading global biopharmaceutical company developing targeted oncology, cardiovascular, and rare disease treatments backed by a strong R&D pipeline. ask →
• KRYSTAL BIOTECH INC (KRYS) Health Care — Krystal Biotech is a genetic medicine company leveraging its proprietary HSV-1 vector platform to treat rare diseases, headlined by its FDA-approved topical gene therapy for dystrophic epidermolysis bullosa. ask →
• CRISPR THERAPEUTICS AG (CRSP) Health Care — CRISPR Therapeutics develops gene-editing medicines using CRISPR/Cas9 technology, with the first approved CRISPR therapy (CASGEVY) for sickle cell disease and beta thalassemia, plus a pipeline of in vivo and cell therapy programs. ask →
• BIOCRYST PHARMACEUTICALS INC (BCRX) Health Care — BioCryst Pharmaceuticals is a biotech company focused on commercializing and developing innovative therapies for hereditary angioedema and other rare diseases using structure-guided drug design. ask →
• AGIOS PHARMACEUTICALS INC (AGIO) Health Care — A commercial-stage biopharmaceutical company focused on transforming the treatment landscape for rare genetic hematologic diseases through its innovative pyruvate kinase activator technology. ask →
• Sarepta Therapeutics (SRPT) Health Care — A biopharmaceutical leader in genetic medicine, specializing in innovative RNA-targeted therapies and gene treatments for Duchenne muscular dystrophy and rare genetic disorders. ask →
• Perrigo (PRGO) Health Care — A struggling diversified consumer and prescription healthcare manufacturer navigating portfolio restructuring and strategic divestitures amid margin pressure and competitive headwinds. ask →
• MONOPAR THERAPEUTICS INC (MNPR) Health Care — Monopar Therapeutics is a clinical-stage biopharmaceutical firm focused on developing innovative radiopharmaceuticals for cancer and specialized treatments for rare diseases like Wilson disease. ask →
• IRONWOOD PHARMA INC CLASS A (IRWD) Health Care — Ironwood Pharmaceuticals is a GI-focused biotech company that generates stable profits from its blockbuster IBS-C therapy, LINZESS, while developing a pipeline for rare intestinal diseases. ask →
• X4 PHARMACEUTICALS INC (XFOR) Health Care — A commercial-stage biopharmaceutical company focused on developing and marketing targeted therapies for rare immune system disorders, headlined by its FDA-approved treatment for WHIM syndrome. 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 →
• PROTARA THERAPEUTICS INC (TARA) Health Care — Clinical-stage biopharmaceutical company developing TARA-002, a cell therapy based on an established immunopotentiator, and IV Choline Chloride for oncology, rare diseases, and nutritional support. ask →
• ATRIUM THERAPEUTICS INC (RNA) Health Care — Atrium is an early-stage biotech pioneering antibody-conjugated RNA therapeutics to treat rare genetic cardiomyopathies by silencing disease-driving genes in the heart. ask →
• TONIX PHARMACEUTICALS HOLDING CORP (TNXP) Health Care — A fully-integrated biopharmaceutical company commercializing TONMYA (cyclobenzaprine sublingual tablets) for fibromyalgia while advancing a pipeline of CNS, immunology, and infectious disease therapies. ask →

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

Last updated 2026-07-22.

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