Alnylam Pharmaceuticals

Alnylam Pharmaceuticals is a global commercial-stage biopharmaceutical company pioneering RNA interference (RNAi) therapeutics — a new class of medicines using small interfering RNA (siRNA) to silence disease-causing genes upstream of protein production. Founded in 2002, the company has delivered six first-in-class approved medicines and launched its "Alnylam 2030" strategy in early 2026 targeting TTR franchise market leadership, 25%+ revenue CAGR through 2030, ~30% non-GAAP operating margin, 10 tissue delivery types, and 40+ clinical programs.

Approved Products (6)

AMVUTTRA (vutrisiran) is a GalNAc-ESC+ conjugate RNAi therapeutic administered subcutaneously quarterly. It received FDA approval in 2022 for hATTR amyloidosis with polyneuropathy (hATTR-PN) and an FDA sNDA approval in March 2025 for ATTR cardiomyopathy (ATTR-CM). The EC approved AMVUTTRA for ATTR-CM in June 2025, with additional approvals in Japan, UK, Brazil, and Canada. ONPATTRO (patisiran) is the first-ever FDA-approved RNAi therapeutic (2018), administered IV via lipid nanoparticle (LNP), approved for hATTR-PN in the...