Alnylam Pharmaceuticals

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Alnylam Pharmaceuticals

Alnylam Pharmaceuticals is a biopharmaceutical company focused on the development of RNA interference (RNAi) therapeutics. The company is headquartered in Cambridge, Massachusetts, and it was founded in 2002. Alnylam is a leader in the field of RNAi, a biological process in which RNA molecules inhibit gene expression or translation, effectively silencing targeted genes.

History

Alnylam Pharmaceuticals was founded in 2002 by a group of scientists and entrepreneurs, including Phillip Sharp, a Nobel laureate in Physiology or Medicine. The company was established to explore the potential of RNA interference as a new class of medicines. In its early years, Alnylam focused on building a robust intellectual property portfolio and establishing collaborations with other pharmaceutical companies.

In 2018, Alnylam received its first FDA approval for an RNAi therapeutic, patisiran, which is used to treat hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) in adults. This marked a significant milestone for the company and the field of RNAi therapeutics.

Technology

Alnylam's core technology is based on RNA interference, a natural cellular process that can selectively silence specific genes. The company utilizes small interfering RNA (siRNA) molecules to target and degrade messenger RNA (mRNA) molecules, preventing the production of disease-causing proteins.

The development of RNAi therapeutics involves several key steps, including the design of siRNA molecules, delivery to target tissues, and ensuring stability and efficacy in the human body. Alnylam has developed proprietary delivery technologies, such as lipid nanoparticles, to enhance the delivery of siRNA to specific tissues.

Products

Alnylam has developed several RNAi-based therapeutics, with a focus on rare genetic diseases, liver diseases, and cardiovascular diseases. Some of the company's notable products include:

• Patisiran: Approved for the treatment of hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis).
• Givosiran: Approved for the treatment of acute hepatic porphyria (AHP).
• Lumasiran: Approved for the treatment of primary hyperoxaluria type 1 (PH1).
• Inclisiran: Developed for the treatment of hypercholesterolemia, in collaboration with The Medicines Company.

Research and Development

Alnylam continues to invest in research and development to expand its pipeline of RNAi therapeutics. The company is exploring new therapeutic areas, including central nervous system disorders and infectious diseases. Alnylam's research efforts are supported by collaborations with academic institutions and other pharmaceutical companies.

Collaborations

Alnylam has established numerous collaborations with other pharmaceutical companies to advance the development and commercialization of RNAi therapeutics. Notable partners include Sanofi, Regeneron Pharmaceuticals, and Vir Biotechnology. These collaborations provide Alnylam with additional resources and expertise to accelerate the development of its pipeline.

Related pages

• RNA interference
• Biopharmaceutical
• Cambridge, Massachusetts
• FDA