Rare Disease Research companies

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…

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…

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

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

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

Philanthropic foundation supporting mathematics and basic science research through grantmaking, computational research, and open-source software development.

Worldwide Clinical Trials is a global Contract Research Organization advancing clinical research across all phases of drug development.