ALNYLAM PHARMACEUTICALS, INC.

Alnylam Pharmaceuticals, Inc. (NASDAQ: ALNY) is a global commercial-stage biopharmaceutical company pioneering the development and commercialization of ribonucleic acid interference, or RNAi, therapeutics. Founded in 2002, Alnylam translates RNAi science into innovative medicines that silence disease-causing genes by targeting messenger RNA before proteins are produced. The company's website is www.alnylam.com.

Alnylam has achieved a significant milestone by bringing six first-in-class RNAi-based medicines to market: AMVUTTRA (vutrisiran), ONPATTRO (patisiran), GIVLAARI (givosiran), OXLUMO (lumasiran), Leqvio (inclisiran, commercialized by Novartis), and Qfitlia (fitusiran, commercialized by Sanofi). These approved products address rare and prevalent diseases across multiple therapeutic areas including transthyretin amyloidosis, acute hepatic porphyria, primary hyperoxaluria type 1, hypercholesterolemia, and hemophilia.

The company operates a robust pipeline of more than twenty clinical programs spanning late-stage and early-stage development. Notable investigational programs include nucresiran for ATTR amyloidosis, zilebesiran for hypertension developed in collaboration with Roche, mivelsiran for Alzheimer's disease and cerebral amyloid angiopathy, and cemdisiran for complement-mediated diseases developed with Regeneron.

Alnylam's technology platform encompasses multiple delivery approaches optimized for different tissues. The company utilizes N-acetylgalactosamine (GalNAc) conjugates for hepatic delivery, lipid nanoparticles for systemic delivery, and hexadecyl (C16) conjugates for central nervous system and ocular delivery. Advanced platforms including ESC+, IKARIA, and GEMINI technologies enable enhanced potency, durability, and multi-target capabilities.

The company pursues a disciplined research strategy targeting genetically validated genes with high unmet medical need, early biomarkers, and clear development pathways. Alnylam has established strategic collaborations with major pharmaceutical companies including Novartis, Sanofi, Roche, and Regeneron to co-develop and commercialize select programs globally. In 2026, the company launched its Alnylam 2030 strategy, aspiring to achieve TTR market leadership, deliver multiple blockbuster medicines beyond TTR, expand to ten tissue types, and achieve sustained profitable growth through disciplined innovation and commercial execution.