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Hemophilia Bleeding Disorder Industry News
Industry News

uniQure Announces Hemophilia B Gene Therapy Program To Enter Pivotal Study

Note: The followed is excerpted from a press release from uniQure. Read uniQure’s full press release HERE.  uniQure announced today plans to advance their development program for hemophilia B gene therapy into a pivotal study using an enhanced version of their product candidate called AMT-061. AMT-061 is identical to AMT-060 in every way apart from a change from […]

Industry News

Bayer Issues Voluntary Recall of Kogenate FS

Note: The following is the full text of an email communication received by HFA from Bayer HealthCare Pharmaceuticals: Bayer in consultation with the FDA has voluntarily recalled approximately 300 vials (part of one lot) of Kogenate® FS, Antihemophilic Factor (Recombinant) in the U.S. as an excipient, used to stabilize the product during manufacturing, may not conform […]

Industry News

Bioverativ Enters Joint Project With UK-Based Bicycle Therapeutics

  Bioverativ and a UK-based biotech company called Bicycle Therapeutics will work together to try to identify new therapies for hemophilia and also sickle cell disease.  The research will focus on using a new approach – called Bicycles – which the companies hope will lead to a new way to treat the blood disorders. Read […]

Industry News

Alnylam Suspends Fitusiran Dosing due to Thrombotic Event

The following is an edited version of a press release from Alnylam Pharmaceuticals. To read the press in full, click here. Alnylam recently became aware of a fatal serious adverse event (SAE) that occurred in a patient with hemophilia A who was receiving fitusiran in the Phase 2 OLE study. As a result, the Company […]

Industry News

FDA Grants Priority Review to Genentech’s Emicizumab for Hemophilia A with Inhibitors

Note: The following is an edited version of a press release from Genentech. Read the press release from Genentech in it’s entirety here. Genentech, a member of the Roche Group, announced today that the U.S. Food and Drug Administration (FDA) has accepted the company’s Biologics License Application (BLA) and granted Priority Review for emicizumab prophylaxis (preventative) as […]

Industry News

Octapharma USA Announces FDA Approval of NUWIQ New Product Strengths

Note: The following is an edited version of a press release from Octapharma USA. Read the press release from Octapharma USA in it’s entirety here. Octapharma USA today announced the U.S. Food and Drug Administration (FDA) has approved new product strengths for NUWIQ. The agency approved new single dose NUWIQ vial strengths of 2500, 3000 and 4000 International […]

Industry News

Spark Therapeutics Releases Preliminary Data for SPK-8011

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. The company has disclosed preliminary initial data from the Phase 1/2 dose-escalation clinical trial for SPK-8011, a novel bio-engineered adeno-associated viral (AAV) vector utilizing the Spark200 capsid and containing a codon-optimized human factor […]

Industry News

Spark Therapeutics Presents Updated Interim Hemophilia B Data Supporting Consistent & Sustained Response at ISTH 2017

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 recently announced that 10 participants in its ongoing Phase 1/2 clinical trial of SPK-9001 for hemophilia B, as of the June 5, 2017 data cut off, had their AIR reduced approximately […]

Industry News

BioMarin’s Investigational Gene Therapy for Hemophilia A Maintains Average Factor VIII Levels within Normal Range for over One Year

Note: The following is an edited version of a press release from BioMarin. Read the press release from BioMarin in it’s entirety here. BioMarin Pharmaceutical Inc. announced an update to its previously reported interim results of an open-label Phase 1/2 study of BMN 270, an investigational gene therapy treatment for severe hemophilia A. The updated results were presented […]

Industry News

uniQure Announces Updated, Long-Term Clinical Data from Ongoing Phase I/II Trial of AMT-060 In Patients with Severe Hemophilia B

Note: The following is an edited version of a press release from uniQure. Read the press release from uniQure in it’s entirety here. uniQure N.V. has announced updated results from its ongoing, dose-ranging Phase I/II trial of AMT-060, its proprietary, investigational gene therapy in patients with severe hemophilia B. The data includes up to 18 months of follow-up from the low-dose […]

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