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About Alnylam PharmaceuticalsAlnylam is leading the translation of RNAi as a new class of innovative medicines, with a core focus on RNAi therapeutics toward genetically defined targets for the treatment of serious, life-threatening diseases with limited treatment options for patients and their caregivers.
RNAi (RNA interference) is a revolution in biology, representing a breakthrough in understanding how genes are turned on and off in cells, and a completely new approach to drug discovery and development. Its discovery has been heralded as "a major scientific breakthrough that happens once every decade or so," and represents one of the most promising and rapidly advancing frontiers in biology and drug discovery today which was awarded the 2006 Nobel Prize for Physiology or Medicine. RNAi is a natural process of gene silencing that occurs in organisms ranging from plants to mammals. By harnessing the natural biological process of RNAi occurring in our cells, the creation of a major new class of medicines, known as RNAi therapeutics, is on the horizon. Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylam's RNAi therapeutic platform, target the cause of diseases by potently silencing specific mRNAs, thereby preventing disease-causing proteins from being made. RNAi therapeutics have the potential to treat disease and help patients in a fundamentally new way.
Alnylam is a biopharmaceutical company developing novel therapeutics based on RNA interference, or RNAi. The company is leading the translation of RNAi as a new class of innovative medicines with a core focus on RNAi therapeutics as genetic medicines, including programs as part of the company's "Alnylam 5x15™" product strategy. Alnylam's genetic medicine programs are RNAi therapeutics directed toward genetically defined targets for the treatment of serious, life-threatening diseases with limited treatment options for patients and their caregivers. These include: patisiran (ALN-TTR02), an intravenously delivered RNAi therapeutic targeting transthyretin (TTR) for the treatment of TTR-mediated amyloidosis (ATTR) in patients with familial amyloidotic polyneuropathy (FAP); revusiran (ALN-TTRsc), a subcutaneously delivered RNAi therapeutic targeting TTR for the treatment of ATTR in patients with TTR cardiac amyloidosis, including familial amyloidotic cardiomyopathy (FAC) and senile systemic amyloidosis (SSA); ALN-AT3, an RNAi therapeutic targeting antithrombin (AT) for the treatment of hemophilia and rare bleeding disorders (RBD); ALN-CC5, an RNAi therapeutic targeting complement component C5 for the treatment of complement-mediated diseases; ALN-AS1, an RNAi therapeutic targeting aminolevulinic acid synthase-1 (ALAS-1) for the treatment of hepatic porphyrias including acute intermittent porphyria (AIP); ALN-PCS, an RNAi therapeutic targeting PCSK9 for the treatment of hypercholesterolemia; ALN-AAT, an RNAi therapeutic targeting alpha-1 antitrypsin (AAT) for the treatment of AAT deficiency-associated liver disease; ALN-HBV, an RNAi therapeutic targeting the hepatitis B virus (HBV) genome for the treatment of HBV infection; ALN-TMP, an RNAi therapeutic targeting TMPRSS6 for the treatment of beta-thalassemia and iron-overload disorders; ALN-ANG, an RNAi therapeutic targeting angiopoietin-like 3 (ANGPTL3) for the treatment of genetic forms of mixed hyperlipidemia and severe hypertriglyceridemia; ALN-AC3, an RNAi therapeutic targeting apolipoprotein C-III (apoCIII) for the treatment of hypertriglyceridemia; ALN-AGT, an RNAi therapeutic targeting angiotensinogen (AGT) for the treatment of hypertensive disorders of pregnancy (HDP), including preeclampsia; ALN-GO1, an RNAi therapeutic targeting glycolate oxidase (GO) for the treatment of primary hyperoxaluria type 1 (PH1); and other programs yet to be disclosed. As part of its "Alnylam 5x15" strategy, as updated in early 2014, the company expects to have six to seven genetic medicine product candidates in clinical development - including at least two programs in Phase 3 and five to six programs with human proof of concept - by the end of 2015. The company's demonstrated commitment to RNAi therapeutics has enabled it to form major alliances with leading companies including Merck, Medtronic, Novartis, Biogen Idec, Roche, Takeda, Kyowa Hakko Kirin, Cubist, GlaxoSmithKline, Ascletis, Monsanto, and The Medicines Company. In early 2014, Alnylam and Genzyme, a Sanofi company, formed a multi-product geographic alliance on Alnylam's genetic medicine programs in the rare disease field. Specifically, Alnylam will lead development and commercialization of programs in North America and Europe, while Genzyme will develop and commercialize products in the rest of world. In addition, Alnylam and Genzyme will co-develop and co-commercialize ALN-TTRsc in North America and Europe. In March 2014, Alnylam acquired Sirna Therapeutics, a wholly owned subsidiary of Merck. In addition, Alnylam holds an equity position in Regulus Therapeutics Inc., a company focused on discovery, development, and commercialization of microRNA therapeutics. Alnylam scientists and collaborators have published their research on RNAi therapeutics in over 200 peer-reviewed papers, including many in the world's top scientific journals such as Nature, Nature Medicine, Nature Biotechnology, Cell, New England Journal of Medicine, and The Lancet. Founded in 2002, Alnylam maintains headquarters in Cambridge, Massachusetts. For more information, please visit www.alnylam.com.
834 articles with Alnylam Pharmaceuticals
The stock market has been rocky recently, but overall the biotech market appears to have been comparatively stable.
Alnylam Pharmaceuticals Reports Fourth Quarter and Full Year 2017 Financial Results and Highlights Recent Period Activity
At December 31, 2017, Alnylam had cash, cash equivalents and fixed income marketable securities, and restricted investments of $1.73 billion, as compared to $1.09 billion at December 31, 2016.
Alnylam Announces FDA Acceptance of New Drug Application (NDA) and Priority Review Status for Patisiran, an Investigational RNAi Therapeutic for the Treatment of Hereditary ATTR (hATTR) Amyloidosis
Alnylam announced today that the U.S. Food and Drug Administration (FDA) has accepted for filing its New Drug Application (NDA) for patisiran, an investigational RNAi therapeutic targeting transthyretin (TTR) for the treatment of hereditary ATTR (hATTR) amyloidosis.
Alnylam announced today that it will report financial results for the fourth quarter and year ending December 31, 2017 on Thursday, February 8, 2018, before the U.S. financial markets open.
Alnylam Announces EMA Acceptance of MAA for Patisiran for the Treatment of Hereditary ATTR (hATTR...
1/26/2018The filing of the MAA was previously announced on December 18, 2017.
The effort was announced last week by the Henri A. Termeer Tribute Committee.
Some achievements are hard to top.
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All other terms of the 2014 Alnylam-Sanofi Genzyme alliance remain unchanged.
Alnylam Expands Alnylam Act Program to Include No-Charge Third-Party Genetic Testing and Counseling for People at Risk for Acute Hepatic Porphyrias
Alnylam today announced it has expanded the Alnylam Act™ program to include no-charge, third-party genetic testing and counseling for individuals who may carry a gene mutation known to be associated with acute hepatic porphyrias (AHPs)
Sanofi and Alnylam Submit Marketing Authorization Application to the EMA for Patisiran for the Treatment of Hereditary ATTR Amyloidosis
Sanofi Genzyme and Alnylam Pharma announced today the submission of a MAA to EMA for patisiran, an investigational RNAi therapeutic targeting transthyretin for the treatment of adults with hereditary transthyretin-mediated amyloidosis.
Alnylam and Sanofi Submit Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA) for Patisiran for the Treatment of Hereditary ATTR (hATTR) Amyloidosis
Alnylam announced today the submission of a Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA) for patisiran.
The possibility of a tax bill being passed has many investors and analysts forecasting 2018 to be a big year for mergers and acquisitions.
Clinical trial dosing will resume around year-end 2017.
Alnylam Completes Submission of New Drug Application to the FDA for Patisiran for the Treatment of Hereditary ATTR (hATTR) Amyloidosis
The rolling submission began on November 15, 2017 with the nonclinical and chemistry, manufacturing and controls components submitted, and now submission of the clinical data completes the filing.
Boston Business Journal pulled together a list of the 25 biggest gainers in the state for 2017, and five of them are biopharma companies.
Alnylam Announces Expansion of U.S. Orphan Drug Designation for Patisiran to Treatment of Transthyretin-Mediated Amyloidosis
Alnylam announced today the FDA has granted a request to amend the orphan drug designation for patisiran to the treatment of transthyretin-mediated amyloidosis (ATTR amyloidosis).
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The drug developed by Alkermes took a lot of flack but proved effective in a recent study.
The company is weeks away from completing its rolling NDA for Patisiran.