Alny

Alnylam Pharmaceuticals, commonly associated with its NASDAQ ticker ALNY, is an American biotechnology company focused on the development of RNA interference (RNAi) therapeutics. The company is headquartered in Cambridge, Massachusetts, and was established in the early 2000s to translate RNAi science into medicines for genetic and rare diseases.

Alnylam’s core technology centers on RNA interference, a mechanism that uses small interfering RNA to selectively silence disease-causing genes. By targeting genes responsible for harmful proteins, the approach aims to provide targeted, disease-modifying treatments with the potential for durable clinical benefits. The company pursues liver-targeted and other tissue-specific programs designed to treat a range of conditions.

Notable milestones include the first FDA approval of an RNAi therapy in 2018: patisiran, sold under the brand Onpattro, for hereditary transthyretin-mediated amyloidosis. Subsequent approvals have expanded the portfolio to include givosiran (brand Givlaari) for acute hepatic porphyrias and lumasiran (brand Oxlumo) for primary hyperoxaluria type 1, reinforcing Alnylam’s position in RNAi medicines.

Alnylam maintains an active pipeline of additional RNAi candidates addressing various rare genetic diseases and other conditions with unmet medical need. The company engages in collaborations with pharmaceutical and academic partners and operates under regulatory oversight from agencies such as the U.S. FDA and corresponding authorities worldwide. Its ongoing progress depends on clinical trial results, manufacturing capabilities, and patient access to its therapies.