ALNYLAM PHARMACEUTICALS, INC. (ALNY)

Alnylam Pharmaceuticals is a global commercial-stage biopharmaceutical company that develops novel therapeutics based on RNA interference (RNAi), a naturally occurring biological pathway used to silence disease-causing genes by targeting messenger RNA before disease-causing proteins are produced. The company has developed five approved first-in-class RNAi medicines: ONPATTRO (patisiran) and AMVUTTRA (vutrisiran) for hereditary transthyretin-mediated amyloidosis (hATTR) with polyneuropathy; GIVLAARI (givosiran) for acute hepatic porphyria; OXLUMO (lumasiran) for primary hyperoxaluria type 1; and Leqvio (inclisiran) for hypercholesterolemia, which is developed and commercialized by collaborator Novartis. The company employs delivery technologies including lipid nanoparticles (LNP) and N-acetylgalactosamine (GalNAc) conjugates for hepatic delivery, and hexadecyl (C16) conjugate approaches for central nervous system and ocular delivery, with additional platforms in development for heart, muscle, and adipose tissue targeting.

Alnylam's pipeline includes late-stage programs such as vutrisiran for ATTR amyloidosis with cardiomyopathy, fitusiran for hemophilia (partnered with Sanofi), and cemdisiran for complement-mediated diseases (partnered with Regeneron, in combination with pozelimab), as well as earlier-stage candidates including zilebesiran for hypertension (co-developed with Roche under a 2023 collaboration agreement) and ALN-APP for Alzheimer's disease and cerebral amyloid angiopathy. The company's strategic "Alnylam P5x25" plan targets becoming a top-tier biotech by the end of 2025, with goals including six or more marketed products, over 20 clinical programs, sustained revenue growth, and non-GAAP profitability. Alnylam markets its products globally, including the U.S., European Union, Japan, and other territories such as Brazil, Canada, and Switzerland, serving patients with rare, specialty, and select prevalent diseases including hATTR amyloidosis, acute hepatic porphyria, primary hyperoxaluria, and hypercholesterolemia.

The company has established numerous collaborations with major pharmaceutical and life sciences partners, including Regeneron, Roche, Novartis, Sanofi, Vir Biotechnology, Novo Nordisk (via its acquisition of Dicerna), and PeptiDream, leveraging its RNAi expertise and intellectual property to support both development and commercialization of its therapeutics, while also maintaining research partnerships such as with the UK Biobank and Our Future Health to advance genetically validated drug targets.