Alnylam Pharmaceuticals

Alnylam Pharmaceuticals

Alnylam Pharmaceuticals is a biopharmaceutical company focused on the development of RNA interference (RNAi) therapeutics. Founded in 2002, Alnylam is headquartered in Cambridge, Massachusetts. The company 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 by a group of scientists and entrepreneurs, including Phillip Sharp, a Nobel laureate in Physiology or Medicine, and Paul Schimmel, a renowned biochemist. The company was established to commercialize RNAi technology, which was a groundbreaking discovery in the field of genetics and molecular biology.

In 2004, Alnylam went public and began trading on the NASDAQ under the ticker symbol ALNY. Over the years, the company has formed strategic partnerships with major pharmaceutical companies, including Novartis, Roche, and Sanofi, to advance its research and development efforts.

Technology

Alnylam's core technology is based on RNA interference, a natural cellular process that can selectively silence specific genes. This technology has the potential to treat a wide range of diseases by targeting the underlying genetic causes. RNAi therapeutics are designed to harness this process by delivering small interfering RNA (siRNA) molecules into cells, where they bind to messenger RNA (mRNA) and prevent the production of disease-causing proteins.

Products and Pipeline

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 (ONPATTRO) - Approved for the treatment of hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis), a rare genetic disorder.
• Givosiran (GIVLAARI) - Approved for the treatment of acute hepatic porphyria (AHP), a group of rare genetic disorders affecting the liver.
• Lumasiran (OXLUMO) - Approved for the treatment of primary hyperoxaluria type 1 (PH1), a rare genetic disorder that results in the overproduction of oxalate.

Alnylam's pipeline includes several other investigational RNAi therapeutics targeting diseases such as hemophilia, hypercholesterolemia, and non-alcoholic steatohepatitis (NASH).

Research and Development

Alnylam invests heavily in research and development to advance its RNAi platform and expand its therapeutic pipeline. The company collaborates with academic institutions and other biotechnology firms to explore new applications of RNAi technology. Alnylam's research efforts are focused on improving the delivery of siRNA molecules to target tissues and enhancing the stability and efficacy of its therapeutics.

Partnerships and Collaborations

Alnylam has established numerous partnerships with leading pharmaceutical companies to co-develop and commercialize its RNAi therapeutics. These collaborations provide Alnylam with additional resources and expertise to accelerate the development of its products and expand its global reach.

Awards and Recognition

Alnylam has received several awards and accolades for its pioneering work in RNAi therapeutics. The company has been recognized for its innovative approach to drug development and its contributions to the field of genetic medicine.

Also see

• RNA interference
• Biopharmaceutical company
• Genetic disorder
• Cambridge, Massachusetts