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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.
896 articles with Alnylam Pharmaceuticals
According to BioPharmCatalyst, there have been (or soon will be) 58 biopharma initial public offerings (IPOs) in 2018, and there’s still more than three months to go.
Alnylam Announces Publication in Circulation of Exploratory Cardiac Endpoint Data from APOLLO Phase 3 Study of Patisiran
Published Data From the APOLLO Study Show That Patisiran Improved Markers of Cardiomyopathy in Patients with Hereditary ATTR Amyloidosis with Polyneuropathy –
Alnylam Investigation: Bernstein Liebhard LLP Announces Investigation Of Alnylam Pharmaceuticals, Inc. - ALNY
Bernstein Liebhard LLP is investigating potential securities fraud claims on behalf of shareholders of Alnylam Pharmaceuticals, Inc.
SHAREHOLDER ALERT: Pomerantz Law Firm Investigates Claims On Behalf of Investors of Alnylam Pharmaceuticals, Inc. - ALNY
Pomerantz LLP is investigating claims on behalf of investors of Alnylam Pharmaceuticals, Inc.
SHAREHOLDER ALERT: Bronstein, Gewirtz & Grossman, LLC Announces Investigation of Alnylam Pharmaceuticals, Inc. (ALNY)
Bronstein, Gewirtz & Grossman, LLC is investigating potential claims on behalf of purchasers of Alnylam Pharmaceuticals, Inc.
Celgene Vet Jackie Fouse Taking Over as CEO of Agios Pharmaceuticals; Companies Also Terminate Pr...
9/5/2018One year after stepping down from her role as president and chief operating officer of Celgene, Jacqualyn (Jackie) Fouse has taken over the reins of Cambridge, Mass.-based Agios Pharmaceuticals.
The biotech industry is fast-moving and dynamic, and staying on top of all the news can be hard for our busy readers. Here’s a look back at some of the top stories you may have missed this month.
8/31/2018The U.S. Food and Drug Administration has had a busy August. In addition to the number of draft guidance documents the agency issued during the month, the FDA has also been busy approving a number of new treatments for a variety of diseases.
ONPATTRO Indicated for the Treatment of Hereditary Transthyretin-Mediated (hATTR) Amyloidosis (hATTR amyloidosis) in Adults with Stage 1 or Stage 2 Polyneuropathy
Alnylam Pharmaceuticals, Inc., the leading RNAi therapeutics company, announced today that management will present company overviews at the following conferences
There’s been another milestone in the history of biotech: The world’s first RNAi gene-silencing drug, Alnylam Pharmaceuticals’ Onpattro, got approved on August 10. That makes 2018 a landmark year. But another year—2014—may prove just as influential for the field and how it develops.
Alnylam Completes Enrollment in ENVISION Phase 3 Study of Givosiran, an Investigational RNAi Therapeutic for the Treatment of Acute Hepatic Porphyrias (AHPs)
Enrollment Completed Ahead of Schedule, Eight Months After First Patient Randomized
Continues Collaboration to Provide Regulatory Submission Support for Additional Patisiran Submissions
It’s not easy to predict trends in drugs, especially with breakthroughs in immunology and genetic engineering often causing dramatic changes in how biopharma companies approach new drugs.
US Bioservices Selected by Alnylam Pharmaceuticals as a Specialty Pharmacy Partner for ONPATTRO™ (patisiran)
Specialty pharmacy to support bringing new FDA-approved medicine to patients
Orsini Healthcare Specialty Pharmacy Selected by Alnylam Pharmaceuticals to Distribute ONPATTRO™ (patisiran)
Orsini Healthcare Specialty Pharmacy announced that it has been selected by Alnylam Pharmaceuticals, Inc to distribute ONPATTRO™ (patisiran) lipid complex injection.
The U.S. Food and Drug Administration (FDA) approved Alnylam Pharmaceuticals’ Onpattro (patisiran) for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults. It is the first and only RNA interference (RNAi) therapeutic to ever be approved.
Alnylam Announces Alignment on Value-Based Agreements with Leading Health Insurers and Launches Comprehensive Patient Support Services for ONPATTRO™ (patisiran)
Structure of Value-Based Arrangements Agreed in Principle with Harvard Pilgrim Health Care and Other Major Health Insurers
Alnylam Announces First-Ever FDA Approval of an RNAi Therapeutic, ONPATTRO™ (patisiran) for the Treatment of the Polyneuropathy of Hereditary Transthyretin-Mediated Amyloidosis in Adults
First and Only FDA-approved Treatment Available in the United States for this Indication
Here’s a roundup of biopharma companies that have announced hiring trends since the beginning of the year.