Alnylam Pharmaceuticals
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Alnylam Pharmaceuticals Logo | |
| Public | |
| Traded as | NASDAQ: ALNY |
| Industry | Pharmaceutical |
| Founded | 2002 |
| Headquarters | 300 Third Street, 3rd Floor, Cambridge, MA, United States |
Key people |
John Maraganore (CEO) Barry Greene (President & COO) Akshay Vaishnaw (CMO) Laurence Reid (CBO) |
| Website |
Alnylam |
Alnylam Pharmaceuticals is a biopharmaceutical company headquartered in Cambridge, Massachusetts. The company’s core focus is the development and commercialization of novel therapeutics based on RNA interference, or RNAi, for genetically defined diseases. Alnylam was founded in 2002 by Phillip Sharp, Paul Schimmel, Dave Bartel, Thomas Tuschl and Phillip Zamore.
RNAi Explained
RNAi is a natural mechanism for silencing specific genes. Genes provide cells with the instructions for making proteins, and proteins — or more specifically proteins made abnormally — are the cause of many human diseases. When a gene is silenced, the cell stops making the protein specified by that gene, thereby improving the course of the disease. RNAi was first discovered in worms in 1998.[1]
In 2001, Alnylam founders began using small interfering RNAs, known as siRNAs, to silence genes in mammalian cells.[2] Human proof of concept has been demonstrated by Alnylam, including in a Phase I trial which resulted in statistically significant reduction of a protein called transthyretin, or TTR.[3] Alnylam has demonstrated human efficacy with intravenous and subcutaneous modes of administration.[4]
Products in development
As of August, 2013, Alnylam had eight therapeutics in clinical and pre-clinical development. Alnylam is currently developing the following therapeutics for genetically defined, life-threatening diseases with limited treatment options (conditions listed below are highly generalized; see each article for more detail):
- ALN-TTR02, targeting transthyretin (TTR) for the treatment of TTR-mediated amyloidosis (ATTR) in patients with familial amyloidotic polyneuropathy (FAP)
- ALN-TTRsc, targeting TTR for the treatment of ATTR in patients with familial amyloidotic cardiomyopathy (FAC);
- ALN-AT3 targeting antithrombin (AT) for the treatment of hemophilia and rare bleeding disorders (RBD);
- ALN-AS1 targeting aminolevulinate synthase-1 (ALAS-1)[5] for the treatment of porphyria including acute intermittent porphyria (AIP)
- ALN-CC5 targeting complement component 5 (C5) for the treatment of complement-mediated diseases
- ALN-PCS, targeting PCSK9 for the treatment of hypercholesterolemia
- ALN-TMP, targeting TMPRSS6 for the treatment of beta-thalassemia and iron-overload disorders
- ALN-AAT, targeting alpha-1-antitrypsin (AAT) for the treatment of AAT deficiency liver disease
Company alliances
Alnylam has formed major alliances with several companies in the pharmaceutical and biotechnology space, including Merck, Medtronic, Novartis, Biogen Idec, Roche, Takeda, Kyowa Hakko Kirin, Cubist, Ascletis, Monsanto, Genzyme, GlaxoSmithKline, and The Medicines Company. In addition, Alnylam and Isis co-founded Regulus Therapeutics, a company focused on discovery, development, and commercialization of microRNA therapeutics; Regulus has formed partnerships with GlaxoSmithKline and Sanofi.
References
- ↑ "Potent and specific genetic interference by double stranded RNA in Caenorhabditis elegans". FIRE, A.; XU, S.; MONTGOMERY, M.K.; KOSTAS, S.A.; DRIVER, S.E. and MELLO, C.C. , Nature, 1998, vol. 391, no. 6669, p. 806-811.
- ↑ "Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells" ELBASHIR, S.M., HARBORTH, J., LENDECKEL, W., YALCIN, A., WEBER, K. & TUSCHL, T. (2001). Nature 2001, vol 411, pp: 494-8.
- ↑ Pollack, Andrew. "A Step Forward for RNA Interference". The New York Times. Retrieved 4 September 2013.
- ↑ Fidler, Ben. "Alnylam Shares Boom on Early Data For Subcutaneous RNA Drug". Xconomy. Retrieved 4 September 2013.
- ↑ "ALAS1 aminolevulinate, delta-, synthase 1 [ Homo sapiens (human) ]". National Center for Biotechnology Information. Retrieved 4 September 2013.