Alnylam Pharmaceuticals

Alnylam Pharmaceuticals

220px Alnylam Pharmaceuticals logo
Type	Public
Traded as	NASDAQ: ALNY
Industry	Pharmaceutical
Founded	2002
Headquarters	300 Third St., Floor 3, Cambridge, MA, USA
Key people	John Maraganore, (CEO) Barry Greene, (Pres., COO) Akshay Vaishnaw, (CMO) Laurence Reid, (CBO)
Products	Product Pipeline
Website	Alnylam.com

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 (RNAi) for genetically defined diseases. Alnylam was founded in 2002 by Phillip Sharp, Paul Schimmel, Dave Bartel, Thomas Tuschl and Phillip Zamore.

History

Founding

Alnylam was founded in 2002 by Phillip Sharp, Paul Schimmel, Dave Bartel, Thomas Tuschl and Phillip Zamore.

The company was named after Alnilam, a star in Orion’s belt. The spelling was tweaked, from Alnilam (the star) to Alnylam, to help it stand out to investors, in Google searches, and in trademark filings.

Therapeutic concept

Main article: RNA interference

Most drug discovery focuses on concepts associated with the ability of molecules—either "small" (low molecular weight drugs, typically organic but sometimes inorganic substances), or "large" (high molecular weight biologic drugs, typically proteins)—to make their way to a disease associated target that is present at some fixed concentration within target cells, and to associate with the target, thereby interfering with its function, and remediating the disease at a molecular level. Examples of this include the action of 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 completely novel approach to therapy involves targeting the very production of a target protein required for the development of the disease. In this regard, RNAi was discovered as a natural mechanism for silencing genes; it was first discovered in a species of worm—a nematode, Caenorhabditis elegans—in 1998. Genes encode the information necessary for cells to synthesize proteins, and proteins—more specifically, proteins made abnormally—cause many human diseases. When a mutant gene is silenced, the cell stops making the abnormal protein specified by that gene, thereby potentially improving the course of the disease. In 2001, Alnylam founders began using small interfering RNAs, known as siRNAs, to silence genes in mammalian cells.

Proofs of concept

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. Alnylam has demonstrated human efficacy with intravenous and subcutaneous modes of administration.

Products

As of August, 2013, Alnylam had eight therapeutics for genetically defined, life-threatening diseases with limited treatment options, in clinical and pre-clinical development. These include the following therapeutics:

• 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) 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; and
• ALN-AAT, targeting alpha-1-antitrypsin (AAT), for the treatment of AAT deficiency liver disease.

Affiliated enterprises

In addition, Alnylam and Isis co-founded Regulus Therapeutics, a company focused on discovery, development, and commercialization of microRNA therapeutics.

Alliances

As of this date, Alnylam has formed major alliances with several companies in the pharmaceutical and biotechnology space, including:

• Ascletis;
• Biogen Idec;
• Cubist Pharmaceuticals;
• Genzyme;
• GlaxoSmithKline;
• Hoffmann-La Roche;
• Kyowa Hakko Kirin;
• Medtronic;
• Merck & Co.;
• Monsanto:
• Novartis;
• Takeda; and
• The Medicines Company.

In addition, the Regulus affiliate has formed partnerships with GlaxoSmithKline and Sanofi.

External links

• Alnylam Corporate Website
• Alnylam on ClinicalTrials.gov