Companies in #Hemophilia

Alnylam Pharmaceuticals (NASDAQ: ALNY) is the leading RNAi therapeutics company, with six approved products (Onpattro, Givlaari, Oxlumo, Amvuttra, Qfitlia, and the Leqvio collaboration with Novartis) and a deep pipeline in cardiometabolic, hepatic infectious disease, and CNS indications. Alnylam's approved medicines target transthyretin amyloidosis (Onpattro, Amvuttra), acute hepatic porphyria (Givlaari), primary hyperoxaluria type 1 (Oxlumo), and hemophilia A and B (Qfitlia). Leqvio (inclisiran), licensed to and commercialized by Novartis, targets LDL cholesterol.

Swiss-headquartered plasma products company specializing in coagulation factor therapies (wilate, Nuwiq) for hemophilia and von Willebrand disease, with operations across 40+ countries.