Alnylam Pharmaceuticals, Inc. Closed Now

Introduction
Alnylam Pharmaceuticals is a pioneering American biotechnology company focused on the development of RNA interference (RNAi) therapeutics. Founded in 2002, Alnylam has become a global leader in the field of RNAi, a Nobel Prize-winning discovery that enables the silencing of disease-causing genes. Its medicines target rare genetic, cardiometabolic, hepatic infectious, and central nervous system diseases.

Mission and Vision

• Mission: To transform the lives of patients with innovative RNAi therapeutics.

• Vision: To build a world-class, sustainable biopharmaceutical company leading in genetic medicine.

Products and Services

• Approved RNAi Medicines:

  • Onpattro (patisiran) – for hereditary transthyretin-mediated amyloidosis (hATTR).

  • Givlaari (givosiran) – for acute hepatic porphyria.

  • Oxlumo (lumasiran) – for primary hyperoxaluria type 1.

  • Amvuttra (vutrisiran) – next-generation treatment for ATTR amyloidosis.

• Pipeline Focus: Expanding into cardiometabolic diseases, CNS, infectious diseases, and broader rare conditions.

• Technology Platform: Proprietary RNAi therapeutic platform enabling precision gene silencing.

Growth and Innovation
Alnylam is the first company to bring RNAi therapies from concept to market, proving the viability of gene silencing as a therapeutic approach. With strategic collaborations with Regeneron, Vir Biotechnology, and Novartis, the company is expanding into larger disease areas beyond rare disorders. Its strong pipeline in cardiovascular and metabolic diseases could significantly broaden its global impact.

Global Impact
Alnylam’s RNAi therapies are available in multiple markets, addressing previously untreatable rare diseases. Its pioneering role in RNAi therapeutics positions it as a scientific leader in genetic medicine. Beyond its patient impact, Alnylam also contributes to advancing biotech innovation and expanding the therapeutic landscape for gene-based medicine worldwide.