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Spark Therapeutics Announces Phase 1/2 Gene Therapy Trial for Hemophilia A

Note: The following is an edited version of a press release from Spark Therapeutics. Read the press release from Spark Therapeutics in it’s entirety here. Spark Therapeutics has announced that its Phase 1/2 trial for the investigation of gene therapy for hemophilia A (factor VIII deficiency) has begun. This trial will initially be enrolling participants at the Children’s Hospital […]

Industry News

Bioverativ Officially Spins-Off of Biogen’s Hemophilia Business

Note: The following is an edited version of a press release from Bioverativ. Read the press release from Bioverativ in it’s entirety here. Today, February 1, 2017, Bioverativ launched as an independent, global biotechnology company focused on the discovery, development, and commercialization of innovative therapies for hemophilia and other rare blood disorders. Bioverativ is formed by a spin-off of Biogen’s […]

Industry News

UniQure Receives FDA Breakthrough Designation of AMT-060 Investigational Gene Therapy

______________________________________________________________________________ Note: The following is edited from a press release from UniQure. Read the full press release in it’s entirety here. UniQure announced that AMT-060,  its proprietary, investigational gene therapy in patients with severe hemophilia B, has received Breakthrough Therapy designation by the U.S. Food and Drug Administration (FDA).  This designation is based on results from the […]

Industry News

Shire’s Adynovate Receives Two New FDA Approvals for Treatment of Hemophilia A

Note: The following is edited from a press release from Shire. Read the full press release in it’s entirety here. Shire plc recently announced that the U.S. Food and Drug Administration (FDA) has approved Adynovate, an extended circulating half-life recombinant Factor VIII treatment for hemophilia A, in pediatric patients under 12 years of age. The FDA […]

Industry News

Genentech Meets Primary Endpoint for Phase III Emicizumab Study

______________________________________________________________________________ Note: The following is edited from a press release from Genentech. Read the full press release in it’s entirety here. Genentech, a member of the Roche Group, announced that the primary endpoint has been met for the Phase III HAVEN 1 study evaluating emicizumab prophylaxis in people 12 years of age or older with hemophilia […]

Industry News

UniQure Announces New Data in Phase I/II Clinical Trial

______________________________________________________________________________ Note: The following is edited from a press release from UniQure. Read the full press release in it’s entirety here. UniQure announced new and updated results during the American Society of Hematology (ASH) meeting in relation to its ongoing, dose-ranging Phase I/II trial of AMT-060. AMT-060 is UniQure’s proprietary, investigational gene therapy for patients with severe […]

Industry News

Sangamo Biosciences Announces Plans to File for New Investigational Drug

______________________________________________________________________________ Note: The following is edited from a press release from Sangamo Biosciences. Read the full press release in its entirety here. Sangamo Biosciences presented data during the recent American Society of Hematology (ASH) Annual Meeting regarding preclinical and manufacturing data of SB-525, its gene therapy program for hemophilia A. “We have developed an improved […]

Industry News

Spark Therapeutics Shares Updated Phase I/II Clinical Trial Data

______________________________________________________________________________ Note: The following is edited from a press release from Spark Therapeutics. Read the full press release in it’s entirety here.. Spark Therapeutics and Pfizer announced updated results from the Phase 1/2 study of SPK-9001, an investigational recombinant gene transfer product in hemophilia B, at the 58th American Society of Hematology (ASH) Annual Meeting, […]

Industry News

Alnylam Presents Phase I Clinical Drug Trial Data at ASH 2016

  Note: The following is an excerpt from a press release from Alnylam Pharmaceuticals, Inc. regarding news from their recent presentation at the American Society of Hematology (ASH) Annual Meeting. The original article can be read in its entirety here. ______________________________________________________________________________ Alnylam Pharmaceuticals, Inc. presented data from the phase 1 study of fitusiran at the […]

Industry News

Octapharma Announces Two Hemophilia A Clinical Trials Utilizing Wilate

The following is excerpted from a press release from Octapharma USA. Read the press release in it’s entirety here.  Octapharma USA has announced that WILATE®, von Willebrand Factor/Coagulation Factor VIII Complex (Human) Lyophilized Powder for Solution for Intravenous Injection will be utilized for two important clinical trials for hemophilia A launching in December. Octapharma USA […]

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