Category: Industry News

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 […]

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 hemophilia […]

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 ongoing, dose-ranging […]

HEMA Biologics Receives FDA License for New Recombinant Factor VIIa Product

Note: The following is edited from a press release from PRNewswire.com. Read the full press release in it’s entirety here.HEMA Biologics, LLC announced that the FDA has accepted the Biologics License Application(BLA) for their first potential product for commercialization in the United States.  The acceptance of the BLA for Coagulation Factor VIIa, Recombinant (eptacog beta activated), […]

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 also […]

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 A and […]

Octapharma Presents Phase III NuProtect Data at 2016 ASH Annual Meeting

______________________________________________________________________________Note: The following is edited from a press release from Octapharma. Read the full press release in it’s entirety here.At the recent American Society of Hematology (ASH) Annual Meeting, Octapharma presented interim data from the ongoing phase 3 study, NuProtect. The NuProtect Study examines the immunogenicity, efficacy and safety of treatment with human cell-line derived recombinant FVIII (Nuwiq) in previously […]

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 hemophilia B. […]

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 gene therapy vector […]

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, in San […]