Alnylam Pharmaceuticals, Inc.

Alnylam Pharmaceuticals, Inc. is a global commercial-stage biopharmaceutical company that develops new medicines based on ribonucleic acid interference (RNAi). RNAi is a natural process in cells that silences and regulates gene expression. The company has developed a new class of medicines called RNAi therapeutics, which use small interfering RNA (siRNA) to silence messenger RNA (mRNA) that encodes proteins involved in diseases, thereby preventing these proteins from being made. This approach aims to transform patient care across various disease areas. Alnylam has achieved approval for six first-in-class RNAi-based medicines: AMVUTTRA® (vutrisiran), ONPATTRO® (patisiran), GIVLAARI® (givosiran), OXLUMO® (lumasiran), Leqvio® (inclisiran), and Qfitlia® (fitusiran). The company's research and development strategy targets genetically validated genes linked to human diseases. They use N-acetylgalactosamine (GalNAc) conjugate or lipid nanoparticle (LNP) methods for delivering siRNAs to the liver. For delivery to the central nervous system and the eye, they use a hexadecyl (C16) moiety as a lipophilic ligand. Alnylam is also developing methods for delivering siRNAs to the heart, skeletal muscle, and adipose tissue. Their focus is on clinical indications with high unmet needs, genetically validated targets, early biomarkers for assessing clinical activity in Phase 1 trials, and a clear path for drug development, regulatory approval, patient access, and commercialization. In early 2026, the company launched its Alnylam 2030 strategy.