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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.
929 articles with Alnylam Pharmaceuticals
Alnylam Pharmaceuticals, Inc. announced today that it has resolved all litigation worldwide with Silence Therapeutics.
In 2019, Alnylam Will Continue ONPATTRO™ Global Launch and Advance Late-Stage Pipeline of Wholly Owned and Partnered Programs with: Five Phase 3 Programs; Three Phase 3 Program Readouts; and Two New Drug Application (NDA) Filings
The idea of throwing Big Pharma under the bus in order to protect innovative biotech companies sparked a robust conversation at the seventh annual Forbes Healthcare Summit.
Alnylam Pharmaceuticals, Inc. announced today that it will webcast its upcoming R&D Day, including a live video stream, on the Investors section of the Company’s website, www.alnylam.com, on the morning of December 6, 2018.
Vir Biotechnology Inc. andAlnylam Pharmaceuticals, Inc. (NASDAQ: ALNY) today announced the initiation of a Phase 1/2 study of VIR-2218, a novel, investigational RNA interference (RNAi) therapeutic for the treatment of chronic hepatitis B virus (HBV) infection.
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that management will present company overviews at the following conferences:
Alnylam Presents Updated Phase 1/2 Open-Label Extension (OLE) Results for Givosiran, an Investigational RNAi Therapeutic for the Treatment of Acute Hepatic Porphyria
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that the Company presented updated results from the ongoing Phase 1/2 open-label extension (OLE) study of givosiran, an investigational RNAi therapeutic, targeting aminolevulinic acid synthase 1 (ALAS1) for the treatment of acute hepatic porphyria (AHP).
Boston Business Journal recently created a list of 15 Massachusetts life science companies that in their third-quarter reports, allocated the highest percentage of total costs to research and development. Here’s a look.
Alnylam Pharmaceuticals Reports Third Quarter 2018 Financial Results and Highlights Recent Period Activity
Obtained FDA and EMA Approvals of ONPATTRO™ (patisiran) – the First-Ever RNAi Therapeutic – and Launched in U.S. and EU
Alnylam Pharmaceuticals, Inc. announced today that management will present company overviews at the following conferences:
11/5/2018Biopharma companies make key appointments to fill leadership positions. Let's take a look!
10/31/2018It may not have always felt like it, but biotech has been in a bull market mode over the past two-and-a-half years, ever since the iShares Biotechnology Index (IBB) bottomed out at $80.01 on February 9, 2016.
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that it will report financial results for the third quarter ending September 30, 2018 on Wednesday, November 7, 2018
Invitae expands work with Alnylam Pharmaceuticals to provide access to genetic testing for primary hyperoxaluria
Part of Invitae's network of partnerships, program supports early diagnosis to help patients and their clinicians make more informed decisions about their health
Alnylam Expands Alnylam Act® Program to Improve Diagnosis of Primary Hyperoxaluria Type 1 (PH1) and Aligns with FDA on Trial Design for ILLUMINATE-B Phase 3 Pediatric Study of Lumasiran
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that it has expanded the Alnylam Act® program to include no-charge, third-party genetic testing and counseling for adults and children who may have mutations in alanine-glyoxylate aminotransferase
Alnylam Announces Advocacy for Impact Grants to Inspire Unique Solutions that Address Unmet Needs Affecting the Rare Disease Community
Alnylam’s Advocacy for Impact Grants Will Fund New Projects of Up to $50,000 to Patient Advocacy Groups that Impact the ATTR Amyloidosis, Acute Hepatic Porphyrias and/or Primary Hyperoxaluria Type 1 Communities
Alnylam Announces Plan to Initiate Rolling Submission of a New Drug Application and Pursue Full Approval for Givosiran
Topline Results on Primary Endpoint of Annualized Attack Rate Expected in Early 2019
Dr. Hamburg is Former FDA Commissioner and Internationally Recognized as a Distinguished Authority in Medicine, Regulatory Science and Public Health
Gotham Therapeutics launched in New York with a $54 million Series A financing. The focus is on a subgroup of RNA metabolism called epitranscriptomics. This involves the biochemical modifications of RNA within a cell, which have an impact on gene expression.
The U.S. Food and Drug Administration gave Akcea Therapeutics and its parent company Ionis Pharmaceuticals the green light for Tegsedi, its treatment for polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.