Companies in #Rare Diseases

Ionis Pharmaceuticals is a leader in RNA-targeted therapeutics, discovering and developing antisense oligonucleotide (ASO) drugs for serious diseases.

Kriya Therapeutics, Inc. is a clinical-stage biopharmaceutical company developing AAV-based gene therapies for common chronic diseases of high unmet need, including Type 1 diabetes, obesity, oncology, and rare diseases. Unlike most AAV gene therapy companies that focus on rare diseases with small patient populations, Kriya's strategic differentiation is targeting chronic conditions with much larger patient populations. Kriya operates its own in-house, large-scale GMP manufacturing facilities for gene therapy at its Research Triangle Park (Durham, North Carolina) headquarters — a notable strategic departure from the typical gene therapy CDMO-out-source model. The company was founded in 2019 by Shankar Ramaswamy, M.D. (former senior partner at McKinsey and former CEO of Axovant) and has raised over $600M in venture funding. Kriya was selected in June 2026 for the FDA PreCheck Pilot Program for its Durham, NC manufacturing facility.

Kyowa Kirin Co., Ltd. is a Japanese specialty pharmaceutical and biotechnology company under the Kirin Holdings group, focused on developing innovative antibody-based drugs for cancer, kidney disease, immune system diseases, and rare diseases. The company is headquartered in Chiyoda-ku, Tokyo and is a member of the Nikkei 225 stock index. Kyowa Kirin is the Japan-based parent of Kyowa Kirin, Inc. (the US subsidiary), and operates as a research-driven biopharma with multiple approved biologics including poteligeo (mogamulizumab, for cutaneous T-cell lymphoma) and Crysvita (burosumab, for X-linked hypophosphatemia). The company announced a $530M biologics manufacturing facility in Sanford, North Carolina in June 2024 — its first US manufacturing site — which was selected in June 2026 for the FDA PreCheck Pilot Program. Kyowa Kirin's US subsidiary (KKNA) operates the Sanford facility and supports the company's clinical and commercial biologics pipeline including rocatinlimab for atopic dermatitis.

Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) is an American biopharmaceutical company founded in 1988 by Leonard Schleifer (current CEO) and George Yancopoulos (current Chief Scientific Officer) and headquartered in Tarrytown, New York. Regeneron is one of the most successful mid-cap-to-large-cap biotechs in the industry, with a portfolio of approved biologics including Eylea (aflibercept, for wet age-related macular degeneration — Regeneron's flagship product), Dupixent (dupilumab, partnered with Sanofi for atopic dermatitis and other Type 2 inflammatory diseases), Libtayo (cemiplimab, partnered with Sanofi for cutaneous squamous cell carcinoma), and Praluent (alirocumab, partnered with Sanofi for LDL cholesterol reduction). Regeneron's pipeline includes multiple bispecific antibodies (linvoseltamab for multiple myeloma, odronextamab for B-cell lymphomas) and the antibodycocktail REGN-COV (casirivimab/imdevimab for COVID-19, now largely superseded by newer variants). The company has committed $7B+ in manufacturing investments across its New York and North Carolina facilities, with a $2B investment in a new Saratoga Springs, NY manufacturing plant announced in 2025 and selected for the FDA PreCheck Pilot Program in June 2026.

REGENXBIO is a clinical-stage biotechnology company developing gene therapies using its proprietary AAV (adeno-associated virus) NAV technology platform. The company is advancing a pipeline of one-time gene therapies for rare and serious diseases including Duchenne muscular dystrophy (RGX-202), Hunter syndrome (RGX-121), and wet age-related macular degeneration (RGX-314, partnered with AbbVie).

Sarepta Therapeutics is a commercial-stage biopharmaceutical company focused on precision genetic medicines for rare neuromuscular and central nervous system diseases.

Scribe Therapeutics is a clinical-stage gene editing company developing next-generation CRISPR-based therapies for serious genetic diseases.

United Therapeutics is a biotechnology company focused on developing therapies for pulmonary arterial hypertension and other rare diseases.

Vertex Pharmaceuticals is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. Vertex is the leading developer of cystic fibrosis therapies (Trikafta/Kaftrio franchise), brought the first FDA-approved CRISPR-based therapy to market (Casgevy, with CRISPR Therapeutics), and has a late-stage pipeline in acute pain, sickle cell disease, beta thalassemia, and Duchenne muscular dystrophy.

Viridian Therapeutics is a clinical-stage biopharmaceutical company developing engineered monoclonal antibodies for autoimmune diseases and rare conditions, with a focus on FcRn inhibitors and thyroid eye disease.