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Alnylam Pharmaceuticals Inc ALNY Stock Quote

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Alnylam Reports Positive Phase 3 Results for Patisiran; Raising FVE on Improved Outlook

Rachel Elfman Equity Analyst

Business Strategy and Outlook

| Rachel Elfman |

Alnylam Pharmaceuticals is a pioneer in designing therapeutics based on RNA interference, or RNAi, a mechanism that occurs naturally in cells and silences overexpressed genes, which often cause protein misfolding and/or protein accumulation. Alnylam seeks to capitalize on the therapeutic potential of RNAi by creating small interfering RNA, or siRNA, that can treat diseases at the genetic level. When siRNA reaches its intended target, it can silence the gene that it contains code for, making it a potentially game-changing technology for difficult to treat diseases caused by genetic mutations.

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Business Description

Alnylam Pharmaceuticals is a leader in the study of RNA interference (RNAi) therapeutics. RNAi is a naturally occurring biological pathway within cells for sequence-specific silencing and regulation of gene expression. Alnylam has five drugs on the market: Onpattro and Amvuttra for hATTR amyloidosis, Givlaari for acute hepatic porphyria, Oxlumo for primary hyperoxaluria type 1, and Leqvio for hypercholesterolemia. It also has several clinical programs across various therapeutic areas: genetic medicines; cardio-metabolic diseases; hepatic infectious diseases, and CNS/ocular diseases. Upfront fees from research partnerships have boosted Alnylam's cash levels, and the company stands to see additional milestones and royalties from drugs commercialized under collaboration partnerships.

Contact
675 West Kendall Street, Henri A. Termeer Square
Cambridge, MA, 02142
T +1 617 551-8200
Industry Biotechnology
Most Recent Earnings Jun 30, 2022
Fiscal Year End Dec 31, 2022
Employees 1,665

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