Alnylam Pharmaceuticals

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Alnylam Pharmaceuticals
Industry Pharmaceutical
Founded 2002
Headquarters 300 Third St., Floor 3, Cambridge, MA, USA
Key people
John Maraganore, (CEO)
Barry Greene, (President, COO)
Akshay Vaishnaw, (CMO)
David-Alexandre Gros (CBO)
Number of employees
> 500 (2017)

Alnylam Pharmaceuticals is a biopharmaceutical company focused on the discovery, development and commercialization of RNA interference (RNAi) therapeutics for genetically defined diseases. The company was founded in 2002 and is headquartered in Cambridge, Massachusetts. In 2016, Forbes included the company on its "100 Most Innovative Growth Companies" list.[1]


In 2002, Alnylam was founded by scientists Phillip Sharp, Paul Schimmel, David Bartel, Thomas Tuschl, and Phillip Zamore, and by investors Christoph Westphal and John Kennedy Clarke; John Maraganore was the founding CEO.[1][2][3][4] The company was named after Alnilam, a star in Orion’s belt. The spelling was modified to make it unique.[5] In 2003, Alnylam merged with the German pharmaceutical company, Ribopharma AG. The newly formed company also received $24.6 million in funding from private-equity firms.[6][7] On February 27, 2004, Alnylam Pharmaceuticals filed for an IPO.[3][8] The company raised $26 million and began trading as ALNY on the Nasdaq stock exchange.[9]

In 2005, the company partnered with Medtronic to develop drug-device combinations to treat neurodegenerative disorders.[10] Alnylam partnered with Biogen Idec to discover and develop potential treatments of progressive multifocal leukoencephalopathy in 2006.[11] In July 2007, Alnylam entered into a nonexclusive alliance with the Swiss drug company, Hoffmann-La Roche, in which Alnylam received $331 million upfront in exchange for access to its technology platform.[12] In September 2007, Alnylam partnered with Isis Pharmaceuticals to found the company Regulus Therapeutics, a company focused on discovery, development, and commercialization of microRNA therapeutics.[13]

In 2009, Alnylam formed alliances with Cubist Pharmaceuticals and Kyowa Hakko Kirin to market a drug developed to treat the respiratory syncytial virus.[14] Alnylam expanded its previous collaboration with Medtronic to include the CHDI Foundation in its Huntington's Disease focused research in 2010.[15] In 2011, Alnylam and GlaxoSmithKline partnered together to develop RNAi technology enhancing vaccine production.[16][17] Alnylam entered into a 10-year alliance with Monsanto in 2012, in order to develop biotech solutions for the farming industry by using natural molecules and developing them for use in crop protection.[18][19][20] In October 2012, the company formed a partnership with Sanofi Genzyme to develop and commercialize a treatment for transthyretin-mediated amyloidosis, a hereditary disease in Asia.[21] In February 2013, Alnylam formed a partnership with The Medicines Company to develop and commercialize a drug to treat a genetic form of high cholesterol.[22]

In July 2013, during a Phase I trial Alnylam demonstrated statistically significant reduction of a protein called transthyretin, or TTR and demonstrated human efficacy with intravenous and subcutaneous modes of administration.[23] In 2014, Sanofi Genzyme acquired a 12 percent stake in Alnylam and increased its rights to several of the company's drugs for $700 million. In a separate transaction Alnylam announced that it had purchased Merck & Co.'s Sirna Therapeutics, for $25 million cash and $150 million in stock.[24][25] In 2015, the company had $41 million in revenue and a market cap of $5.2 billion.[26]

In 2016, the company purchased land in Norton, Massachusetts to build a manufacturing facility.[27][28]

In October 2016 the Phase III clinical trial of the company's lead product, revusiran, was halted due to increased deaths in the drug arm of the trial, and the company said it was terminating development of the compound.[29][30]

Therapeutic concept[edit]

Most drug discovery seeks molecules which associate with a "target" associated with a disease and interfere with its function, thus remediating the disease at a molecular level. Examples include small molecule HIV protease inhibitors, which are taken orally, and via the bloodstream, make their way to cells infected with the HIV virus, and block a key enzyme, virally encoded enzyme, HIV protease, by occupying its active site, thereby refusing access of a key viral component, and preventing a key step in the HIV virus life cycle (scission of the HIV polyprotein).

A different approach targets the production of a protein required for the development of the disease. RNAi was discovered to be a natural mechanism for silencing genes through cleavage and degradation of mRNA, first discovered in C. elegans in 1998 by Andrew Fire and Craig Mello.[31] Genes encode the information necessary for cells to synthesize proteins, and proteins made abnormally cause many human diseases. When a mutant gene is silenced, the cell stops making the abnormal protein specified by that gene, potentially improving the course of the disease. In 2001, Alnylam founders began using small interfering RNAs, known as siRNAs, to silence genes by targeting mRNA in mammalian cells.[32]


In 2016, Alnylam Pharmaceuticals had 18 potential treatments[33] in various development stages across three Strategic Therapeutic Areas (STArs), Genetic Medicine, Cardio-Metabolic Disease and Hepatic Infectious Disease. In late 2016, the company's lead candidate in phase III studies was patisiran, a treatment targeting transthyretin (TTR) for the treatment of TTR-mediated amyloidosis (ATTR), in patients with familial amyloidotic polyneuropathy (FAP).[29]


  1. ^ a b "Most Innovative Growth Companies #100 Alnylam Pharmaceuticals". Forbes. May 2016. Retrieved September 22, 2016. 
  2. ^ "Alnylam Pharmaceuticals". CrunchBase. Retrieved September 22, 2016. 
  3. ^ a b Hollmer, Mark (March 8, 2004). "Cambridge Bio-Startup Alnylam Files For IPO". Boston Business Journal. Retrieved September 22, 2016. 
  4. ^ "Alnylam S-1". Alnylam via SEC Edgar. February 27, 2004. 
  5. ^ Dolgin, Elie (March 9, 2016). "What's in a Biotech Name? How Drug Startups Choose Their Name". STAT. Retrieved September 22, 2016. 
  6. ^ Naik, Gautam (July 7, 2003). "Ribopharma, Alnylam Announce Merger". The Wall Street Journal. Retrieved September 22, 2016. 
  7. ^ "Alnylam, ribopharma Merge, Notch $24.6 Million in Financing". Boston Business Journal. July 7, 2003. Retrieved September 22, 2016. 
  8. ^ "Alnylam Pharmaceuticals, Inc. (ALNY) IPO". Nasdaq. May 28, 2004. Retrieved September 22, 2016. 
  9. ^ Fodor, Kate (October 11, 2004). "Biotechs Put Initial Public Offerings On Hold". The Scientist. Retrieved September 22, 2016. 
  10. ^ "Medtronic, Alnylam to Seek treatments". Memphis Business Journal. February 9, 2005. Retrieved September 22, 2016. 
  11. ^ "Biogen Idec and Alnylam Partner in RNAi Drug Deal". Boston Business Journal. September 21, 2006. Retrieved September 22, 2016. 
  12. ^ Greil, Anita (July 10, 2007). "Roche Bets on Gene Research". The Wall Street Journal. Retrieved September 22, 2016. 
  13. ^ Hutton, David (June 2011). "Surf's Up For MicroRNAs". Drug Discovery News. Retrieved September 22, 2016. 
  14. ^ Roush, Wade (January 9, 2009). "Alnylam, Cubist Will Cooperate on RNAi Drug for Lung Infections". Xconomy. Retrieved September 22, 2016. 
  15. ^ Taylor, Nick (November 8, 2010). "Alnylam, Medtronic & CHDI Form RNAi Delivery Collaboration". in-Pharma Technologist. Retrieved September 22, 2016. 
  16. ^ Weintraub, Arlene (November 1, 2011). "Alnylam and Glaxo Partner on RNAi for Vaccine Production". Xconomy. Retrieved September 22, 2016. 
  17. ^ "GSK Taps Alnylam Tech For Faster Vaccine Production". Fierce Pharma. November 3, 2011. Retrieved September 22, 2016. 
  18. ^ Weintraub, Arlene (August 28, 2012). "Alnylam Inks $29M deal With Monsanto For New Bio Tools for Farmers". Xconomy. Retrieved September 22, 2016. 
  19. ^ Macron, Doug (September 6, 2012). "Monsanto, Alnylam Form $29M Alliance to Apply RNAi to Agriculture". GenomeWeb. Retrieved September 22, 2016. 
  20. ^ "Alnylam and Monsanto Form Strategic Alliance". BusinessWire. August 28, 2012. Retrieved September 22, 2016. 
  21. ^ Reidy, Chris (October 22, 2012). "Alnylam, Genzyme Form Alliance". The Boston Globe. Retrieved September 22, 2016. 
  22. ^ Pollack, Andrew (February 4, 2013). "Medicines Co. buys Cholesterol Drug Rights". The New York Times. Retrieved September 22, 2016. 
  23. ^ Fidler, Ben (2013-07-11). "Exome: Alnylam Shares Boom on Early Data For Subcutaneous RNA Drug". Xconomy (online). Retrieved 4 September 2013. 
  24. ^ Bennett, Simeon (January 13, 2014). "Sanofi to Pay Alnylam $700 Million for Rare-Disease Drugs". Bloomberg. Retrieved September 22, 2016. 
  25. ^ Stanton, Dan (January 13, 2014). "Alnylam Buys Merck's siRNA Tech Biz as Sanofi Inks $700m Alliance". In-Pharma Technologist. Retrieved September 22, 2016. 
  26. ^ Seiffert, Don (March 22, 2016). "The 9 fastest-growing biotech firms in the Bay State". Boston Business Journal. 
  27. ^ Stanton, Dan (February 17, 2016). "Alnylam Lays Down Plans for Commercial RNAi Manufacturing Plant". In-Pharma Technologist. Retrieved September 22, 2016. 
  28. ^ Weisman, Robert (February 11, 2016). "Alnylam Buys Norton Site for $200M Drug Manufacturing Plant". Boston Globe. Retrieved September 22, 2016. 
  29. ^ a b Adams, Ben (October 6, 2016). "Alnylam plunges after deaths force it to pull PhIII blockbuster candidate revusiran". FierceBiotech. 
  30. ^ "After 18 Deaths in Phase III, Alnylam Halts Revusiran Development". Genetic Engineering and Biotechnology News. October 6, 2016. 
  31. ^ Hopkin, Michael (October 2, 2006). "RNAi Scoops Medical Nobel". Nature. Retrieved September 22, 2016. 
  32. ^ "About RNAi – Alnylam". 
  33. ^ "Alnylam Pharmaceuticals Inc.". BioCentury. Retrieved September 22, 2016. 

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