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 + market data, shown with its as-of date.

• Pfizer (PFE) Health Care — A diversified, global biopharmaceutical giant focused on discovering and commercializing life-changing medicines, vaccines, and advanced therapies across key therapeutic areas. ask →
• Vertex Pharmaceuticals (VRTX) Health Care — Vertex is a leader in biotechnology primarily recognized for its dominance in cystic fibrosis therapy and its emerging pipeline of gene-editing treatments and non-opioid pain relief. ask →
• Moderna (MRNA) Health Care — Moderna is a pioneer in mRNA technology, working to transform medicine by developing a broad pipeline of prophylactic vaccines and therapeutic medicines. ask →
• United Therapeutics (UTHR) Health Care — United Therapeutics is a biotechnology company dedicated to providing innovative, life-extending therapies for patients with pulmonary arterial hypertension and other rare diseases through a focus on proprietary treprostinil-based treatments. ask →
• Jazz Pharmaceuticals (JAZZ) Health Care — Jazz Pharmaceuticals is a biopharmaceutical company dedicated to developing innovative medicines for patients with complex sleep disorders and challenging oncology conditions. ask →
• Corcept Therapeutics (CORT) Health Care — Corcept Therapeutics is a commercial-stage biotechnology company pioneering cortisol modulation therapies to treat severe endocrine, oncologic, and metabolic conditions. ask →
• BioMarin Pharmaceutical (BMRN) Health Care — BioMarin is a biotechnology leader dedicated to transforming the lives of patients with rare, genetically defined diseases through innovative, category-defining medicines. ask →
• 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 →
• Ionis Pharmaceuticals (IONS) Health Care — Ionis Pharmaceuticals is a pioneer in RNA-targeted therapies, transitioning into a commercial-stage biotech with a pipeline focused on neurology and cardiometabolic diseases. ask →
• PTC THERAPEUTICS INC (PTCT) Health Care — A commercial-stage biopharmaceutical company dedicated to discovering and developing innovative therapies for rare genetic diseases. ask →
• TRAVERE THERAPEUTICS INC (TVTX) Health Care — A biopharmaceutical company developing and commercializing innovative therapies to address rare kidney and metabolic diseases with high unmet need. ask →
• ACADIA PHARMACEUTICALS INC (ACAD) Health Care — Acadia Pharmaceuticals is a biopharmaceutical company specializing in developing and commercializing innovative treatments for central nervous system and rare disease indications. ask →
• AMYLYX PHARMACEUTICALS INC (AMLX) Health Care — Amylyx Pharmaceuticals is a clinical-stage biotech developing novel treatments for rare endocrine and neurodegenerative conditions, with a late-stage lead candidate for post-bariatric hypoglycemia. 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 →
• HARMONY BIOSCIENCES HLDG INC (HRMY) Health Care — Harmony Biosciences is a pharmaceutical company focused on delivering innovative therapies for rare neurological disorders, anchored by its commercial success in narcolepsy treatment. ask →
• BIOCRYST PHARMACEUTICALS INC (BCRX) Health Care — BioCryst is a rare-disease-focused biotech company commercializing the once-daily oral HAE therapy ORLADEYO while developing a pipeline of next-generation treatments. ask →
• VIRIDIAN THERAPEUTICS ORS INC (VRDN) Health Care — A biopharmaceutical company commercializing therapies for thyroid eye disease and developing a pipeline of treatments for autoimmune and rare diseases. 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 →
• AGIOS PHARMACEUTICALS INC (AGIO) Health Care — Agios Pharmaceuticals is a rare disease-focused biopharmaceutical company advancing a pipeline of novel hematology and genetic disorder therapies, anchored by its pyruvate kinase activator, mitapivat. ask →
• ETON PHARMACEUTCIALS INC (ETON) Health Care — Eton Pharmaceuticals is a specialty drug company dedicated to acquiring and commercializing a diverse portfolio of therapies for rare diseases. ask →
• Ultragenyx (RARE) Health Care — Ultragenyx is a biopharmaceutical company dedicated to transforming the lives of patients suffering from rare and ultra-rare genetic diseases through innovative therapeutic development. ask →
• OMEROS CORP (OMER) Health Care — A biopharmaceutical company bridging commercial success from its ocular surgery product with a clinical-stage pipeline targeting complement-mediated disorders. ask →
• SAVARA INC (SVRA) Health Care — A clinical-stage biopharmaceutical company dedicated to developing novel treatments for rare respiratory diseases. 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 →
• IRONWOOD PHARMA INC CLASS A (IRWD) Health Care — Ironwood Pharmaceuticals is a GI-focused biotech company commercializing the market-leading treatment for IBS-C and CIC, LINZESS, while advancing its late-stage pipeline asset for short bowel syndrome. 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 →
• CAPRICOR THERAPEUTICS INC (CAPR) Health Care — A clinical-stage biotechnology company developing novel cell and exosome-based therapies for Duchenne muscular dystrophy and other rare diseases. ask →
• TECTONIC THERAPEUTIC INC (TECX) Health Care — A clinical-stage biotechnology company developing GPCR-targeted biologic medicines for diseases with significant unmet medical needs. ask →
• ARCTURUS THERAPEUTICS HOLDINGS INC (ARCT) Health Care — Arcturus Therapeutics is a clinical-stage mRNA company developing liver and respiratory disease therapeutics powered by its proprietary self-amplifying mRNA and lipid nanoparticle delivery platforms. 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 →
• X4 PHARMACEUTICALS INC (XFOR) Health Care — A biopharmaceutical company developing mavorixafor, an oral CXCR4 antagonist, to treat rare hematology conditions like chronic neutropenia and WHIM syndrome. ask →
• VANDA PHARMACEUTICALS INC (VNDA) Health Care — Vanda Pharmaceuticals is a specialty biopharmaceutical company commercializing psychiatric and neurological therapies while advancing a pipeline focused on pharmacogenetic-driven precision medicine. 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 →
• KEROS THERAPEUTICS INC (KROS) Health Care — Keros Therapeutics develops novel protein therapeutics targeting the TGF-ß pathway to address severe neuromuscular, bone, and blood disorders. ask →
• SIONNA THERAPEUTICS INC (SION) Health Care — Sionna Therapeutics is a clinical-stage biopharma company pioneering NBD1-stabilizing therapies to treat the underlying cause of cystic fibrosis. ask →
• PROTALIX BIOTHERAPEUTICS INC (PLX) Health Care — Protalix BioTherapeutics leverages its proprietary plant cell-based protein expression platform to develop and commercialize life-saving enzyme replacement therapies for rare diseases. ask →
• ENTRADA THERAPEUTICS INC (TRDA) Health Care — Entrada Therapeutics is developing a new class of intracellular genetic medicines using its proprietary endosomal escape vehicle technology to treat serious neuromuscular and retinal diseases. ask →
• PROTARA THERAPEUTICS INC (TARA) Health Care — Clinical-stage biopharmaceutical company developing TARA-002 for bladder cancer and rare lymphatic malformations, and IV Choline Chloride for patients on parenteral support. ask →
• ATRIUM THERAPEUTICS INC (RNA) Health Care — Atrium Therapeutics is pioneering precision RNA-based therapies to treat genetically driven cardiomyopathies by targeting disease drivers directly in the heart. ask →
• SPRUCE BIOSCIENCES INC (SPRB) Health Care — A clinical-stage biopharmaceutical company advancing a breakthrough enzyme replacement therapy for the rare, neurodegenerative condition Sanfilippo Syndrome Type B. ask →
• TONIX PHARMACEUTICALS HOLDING CORP (TNXP) Health Care — A commercial-stage biopharmaceutical company focused on CNS, immunology, and infectious disease therapies, headlined by its FDA-approved fibromyalgia treatment, TONMYA. ask →

Not quite it? Describe exactly what you want →

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.