Specialty Pharma Journal Specialty, Precision & Technology
CAMBRIDGE, Mass.–(BUSINESS WIRE)–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). The new …
Tag Archives: RNAi
November, 2018
October, 2018
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30 October
Lilly and Dicerna Announce RNAi Licensing and Research Collaboration
INDIANAPOLIS and CAMBRIDGE, Mass., Oct. 29, 2018 /PRNewswire/ — Eli Lilly and Company (NYSE: LLY) and Dicerna Pharmaceuticals (NASDAQ: DRNA) today announced a global licensing and research collaboration focused on the discovery, development and commercialization of potential new medicines in the areas of cardio-metabolic disease, neurodegeneration and pain. The companies will utilize Dicerna’s proprietary GalXC™ RNAi technology platform …
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24 October
Alexion and Dicerna Collaborate to Discover and Develop RNAi Therapies for Complement-Mediated Diseases
BOSTON & CAMBRIDGE, Mass.–(BUSINESS WIRE)–Alexion Pharmaceuticals, Inc.(NASDAQ:ALXN) and Dicerna Pharmaceuticals, Inc. (NASDAQ:DRNA) today announced a collaboration to discover and develop RNA interference (RNAi) therapies for complement-mediated diseases. An RNAi-based approach to blocking the production of complement pathway factors offers the potential to inhibit the uncontrolled complement activation that leads to many diseases. …
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15 October
Alnylam to Initiate Rolling Submission of NDA and Pursue Full Approval for Givosiran for Acute Hepatic Porphyria
CAMBRIDGE, Mass.–(BUSINESS WIRE)–Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced that in consultation with the U.S. Food and Drug Administration (FDA), the Company plans to pursue a full approval based on the complete results of the ENVISION Phase 3 study of givosiran, an investigational RNAi therapeutic targeting …
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4 October
Arrowhead Enters $3.7 Billion License and Collaboration Agreements with Janssen
PASADENA, Calif.–(BUSINESS WIRE)–Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) today announced that it entered into a license and collaboration agreement with Janssen Pharmaceuticals, Inc., part of the Janssen Pharmaceutical Companies of Johnson & Johnson, to develop and commercialize ARO-HBV. In addition, Arrowhead entered into a research collaboration and option agreement with Janssen …
June, 2018
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8 June
Alnylam Reports Updated Positive Results from Phase 1/2 Study of Lumasiran in Primary Hyperoxaluria Type 1
CAMBRIDGE, Mass.–(BUSINESS WIRE)–Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today new positive results from its Phase 1/2 study with lumasiran, an investigational RNAi therapeutic targeting glycolate oxidase (GO) for the treatment of Primary Hyperoxaluria Type 1 (PH1). Results were presented at the OxalEurope, European Hyperoxaluria Consortium, taking …
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4 June
FDA Grants Orphan Drug Designation to Alnylam’s Investigational RNAi Therapeutic for Treatment of Transthyretin-Mediated Amyloidosis
CAMBRIDGE, Mass.–(BUSINESS WIRE)–Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that the United States Food and Drug Administration (FDA) has granted Orphan Drug Designation to ALN-TTRsc02, an investigational RNAi therapeutic for the treatment of transthyretin-mediated amyloidosis. ALN-TTRsc02 has the potential to be a once-quarterly, low volume, subcutaneously …
December, 2017
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15 December
FDA Lifts Clinical Hold on Alnylam’s Hemophilia Drug Candidate Fitusiran
CAMBRIDGE, Mass. & PARIS–(BUSINESS WIRE)–Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, and Sanofi Genzyme, the specialty care global business unit of Sanofi, announced today that the U.S. Food and Drug Administration (FDA) has lifted the hold on clinical studies with fitusiran, including the Phase 2 open-label extension (OLE) study …
November, 2017
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20 November
FDA Grants Breakthrough Therapy Designation to Alnylam’s Investigational RNAi Drug
CAMBRIDGE, Mass.–(BUSINESS WIRE)–Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) for patisiran, an investigational RNAi therapeutic targeting transthyretin (TTR) for the treatment of adults with hereditary transthyretin-mediated ATTR amyloidosis (hATTR amyloidosis) with polyneuropathy. BTD …
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17 November
Alnylam Starts Rolling FDA Filing for Patisiran for the Treatment of Hereditary ATTR
CAMBRIDGE, Mass.–(BUSINESS WIRE)–Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced the company has initiated submission of a rolling New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for patisiran, an investigational RNAi therapeutic targeting transthyretin (TTR), for the treatment of hereditary ATTR (hATTR) amyloidosis. …