Rare Disease Therapeutics companies

Alnylam Pharmaceuticals is a biopharmaceutical company pioneering RNA interference (RNAi) therapeutics to treat diseases by silencing the genes that cause them.

BioMarin is a global biotechnology company that develops and commercializes therapies for rare genetic diseases.

Biopharmaceutical company developing rare disease therapies focused on cellular metabolism, with FDA-approved treatments and pipeline candidates in hematology.

Harmony Biosciences is a commercial-stage pharmaceutical company developing and commercializing therapies for rare neurological diseases, headquartered in Plymo…

ANI Pharmaceuticals is a US-based biopharmaceutical company that develops, manufactures, and markets branded and generic prescription drugs, with capabilities i…

Structure Therapeutics is a clinical-stage biopharmaceutical company using structure-based drug design to develop oral small molecule therapeutics for chronic m…

Xaira Therapeutics is an integrated biotechnology company driving advances in artificial intelligence to learn the language of life and transform how we treat d…

Frontier Medicines is a precision medicine company developing a platform combining chemoproteomics, covalent drug discovery, and machine learning to target prev…

Tahoe Therapeutics develops a platform generating gigascale single-cell maps of drug-cell interactions for drug discovery, along with large-scale biological dat…

Dyno Therapeutics uses artificial intelligence to engineer improved capsid proteins for gene therapy delivery, operating in the biotechnology and genetic medici…

Citizen Health builds an AI-powered personal health advocate for patients with rare and complex diseases, using their own health data to assist with care and re…