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BIOTECHNOLOGY

Alnylam Pharmaceuticals

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Alnylam Pharmaceuticals

BiotechnologyCambridge, United States
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About Alnylam Pharmaceuticals

The company that proved RNA interference could be turned into medicine, Alnylam Pharmaceuticals has built the world's leading RNAi therapeutics pipeline from the ground up. Founded in Cambridge, Massachusetts in 2002, the company was established with a singular mission: to harness the natural gene-silencing mechanism of RNA interference to treat diseases at their genetic root. What was once a Nobel Prize-winning biological discovery has, under Alnylam's stewardship, become a bona fide drug class.

Approved Medicines and the GalNAc Platform

Alnylam's commercial portfolio spans multiple approved products targeting serious, often rare, diseases. ONPATTRO (patisiran), approved in 2018, was the first-ever RNAi therapeutic to reach patients, indicated for hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis). It was followed by GIVLAARI (givosiran) for acute hepatic porphyria, OXLUMO (lumasiran) for primary hyperoxaluria type 1, and LEQVIO (inclisiran, developed with Novartis) for cardiovascular cholesterol management. Central to the commercial and pipeline strategy is the proprietary GalNAc conjugate delivery platform, which enables subcutaneous dosing and has significantly broadened the range of addressable targets in the liver.

Pipeline Reach and Global Presence

Beyond its approved medicines, Alnylam maintains an active investigational pipeline targeting conditions including transthyretin amyloidosis with cardiomyopathy, complement-mediated diseases, and hypertension, among others. The company operates globally, with offices and partnerships spanning North America, Europe, and Asia. Alnylam trades on the Nasdaq Stock Market under the ticker ALNY and is considered a large-cap biotechnology company. Its partnerships with major pharmaceutical companies, including a long-standing collaboration with Sanofi, have reinforced its commercial infrastructure and expanded patient access internationally.

Alnylam's work represents a structural shift in how genetic medicine is approached, moving from managing symptoms to silencing the genes that cause disease in the first place. For medical affairs, licensing, and pipeline enquiries, further information is available at alnylam.com.

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