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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.
863 articles with Alnylam Pharmaceuticals
Alnylam Pharmaceuticals, Inc. announced today that it plans to host its 5th annual series of “RNAi Roundtable” webcasts this summer.
Alnylam to Report New Clinical Results for Patisiran at the 4th Congress of the European Academy of Neurology
The results being presented at EAN add to the wealth of data from the APOLLO Phase 3 study of patisiran that provide evidence for the potential of this investigational RNAi therapeutic for the treatment of hATTR amyloidosis
Over 700 Alnylam Employees Volunteer at the Company’s 4th Annual “Helping Hands” Community Service Day
Alnylam Pharmaceuticals, Inc. kicked off its fourth annual company-wide “Helping Hands” Community Service Day benefiting 23 organizations, including 16 organizations in the Greater Boston area and seven organizations in Western Europe.
Alnylam Reports Updated Positive Results from Phase 1/2 Study of Lumasiran in Patients with Primary Hyperoxaluria Type 1 (PH1)
Patients Receiving Investigational Lumasiran Experienced Substantial and Sustained Reductions in Urinary Oxalate, Confirming Potential for RNAi-Mediated Inhibition of Glycolate Oxidase as a Robust Therapeutic Approach for PH1
Actor, director and advocate Rob Reiner opened the third day of the 2018 BIO International Convention.
The BIO International Convention Begins Day Three with Keynote Speech from Actor, Director and Advocate Rob Reiner
Wednesday programming will emphasize the patient and the role of the biotech industry in delivering care.
Alnylam Receives Orphan Drug Designation from the United States Food & Drug Administration for ALN-TTRsc02, a Subcutaneously Delivered Investigational RNAi Therapeutic for the Treatment of Transthyretin-Mediated Amyloidosis
ALN-TTRsc02 has the potential to be a once-quarterly, low volume, subcutaneously administered RNAi medication in the management of this serious disease.
Madrigal Pharmaceuticals announced positive topline data from its Phase II clinical trial of MGL-3196 in patients with biopsy-proven non-alcoholic steatohepatitis (NASH).
Most investors thought 2018 would be a big year for mergers and acquisitions in the biopharma industry because of changes to the tax law, and so far they’ve been right.
coreHEM Publishes Core Outcome Set for Hemophilia Clinical Trials; Prepares to Launch Next Phase of Work
The coreHEM initiative has announced the publication of a core outcome set for clinical trials of gene therapy in hemophilia.
Pharma and biotech companies across the globe continued to expand and reshape their leadership teams with new hires this past week. BioSpace collected several announced leadership appointments, which includes new chief executive officers, new members to boards of directors and more.
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today the appointment of a veteran healthcare leader, Colleen Reitan, to Alnylam’s Board of Directors, effective June 1, 2018.
Alnylam Pharmceuticals reported preclinical results that supports advancement of its RNA interference (RNAi) therapeutics for central nervous system disorders into the clinic.
Alnylam Pharmaceuticals Reports First Quarter 2018 Financial Results and Highlights Recent Period Activity
– Presented Positive New Clinical Results from APOLLO Phase 3 Study of Patisiran, and Phase 1 and Phase 1/2 Open-Label Extension (OLE) Studies of Givosiran – – Advanced Two Additional RNAi Therapeutics in Phase 3 Development: Inclisiran in ORION-9, -10, and -11 Studies, and Fitusiran in the ATLAS Program –
Alnylam Achieves Alignment with FDA on Accelerated Development Path for Lumasiran, an Investigational RNAi Therapeutic for the Treatment of Primary Hyperoxaluria Type 1 (PH1)
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that the Company has reached alignment with the U.S. Food and Drug Administration (FDA) on a pivotal study design for lumasiran, an investigational RNAi therapeutic for the treatment of primary hyperoxaluria type 1 (PH1).
Alnylam Pharmaceuticals, Inc. announced that management will present company overviews at the following conferences:
The difference between the salaries paid to top executives and the median income earned by rank and file employees has been a sore subject for many years and has served as a wedge political issue in recent elections.
Alnylam Pharmaceuticals, Inc. announced that it will report financial results for the first quarter ending March 31, 2018 on Thursday, May 3, 2018, after the U.S. financial markets close.
Alnylam Reports New Clinical Results from the APOLLO Phase 3 Study of Patisiran at the American Academy of Neurology 2018 Annual Meeting
Patisiran Reduced the Composite Rate of All-Cause Hospitalization and Mortality by Approximately 50 Percent, Relative to Placebo
Alnylam Receives Positive Opinion for Orphan Drug Designation in the European Union for ALN-TTRsc02, a Subcutaneously Delivered Investigational RNAi Therapeutic for the Treatment of Transthyretin-Mediated Amyloidosis
Designation for the treatment of transthyretin (TTR)-mediated (ATTR) amyloidosis.