Rare Disease companies

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

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

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

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

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…

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

Baylor Genetics provides clinical diagnostic genomics testing including whole genome sequencing, exome sequencing, and RNA analysis to support diagnosis across…