Receive Our Newsletter

Hemophilia Bleeding Disorder Industry News
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 […]

Industry News

Bioverativ Files Complaints Against CSL Behring’s Idelvion

Note: This story has been sourced from the website, www.thepharmaletter.com and The Australian Business Review. On Friday, July 7, 2017, Bioverativ filed complaints in the U.S. District Court for the District of Delaware and with the U.S. International Trade Commission alleging that Idelvion, a novel factor IX albumin fusion protein developed by CSL Behring, infringes upon […]

Industry News

Alnylam & Sanofi Genzyme Report Positive Results from Ongoing Phase 2 Study with Investigational RNAi Therapeutic Fitusiran in Patients with Hemophilia A and B With or Without Inhibitors

Note: The following is an edited version of a press release from Alnylam. Read the press release from Alnylam in it’s entirety here. Alnylam Pharmaceuticals, Inc. and Sanofi Genzyme, announced today new positive results from the ongoing Phase 2 open-label extension study with fitusiran in patients with hemophilia A and B, with or without inhibitors. These results were presented today […]

Industry News

Shire Gets Injunction Against Roche Over Emicizumab

Note: The following is an excerpt from a Reuters news article. Read the article in full here. Pharmaceutical group Shire said on Sunday it had obtained a preliminary injunction in a Hamburg court against rival Roche over its hemophilia drug emicizumab, alleging incomplete and misleading statements surrounding the treatment. Roche said in an emailed statement it […]

Industry News

Genentech Announces Positive Phase III Results for Emicizumab at ISTH 2017

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 data from HAVEN 1, a Phase III study evaluating once-weekly subcutaneous emicizumab prophylaxis (preventative) in adults and adolescents with hemophilia A with inhibitors, were published […]

Industry News

Shire Submits Investigational New Drug Application to FDA for Gene Therapy candidate SHP654 for treatment of Hemophilia A

Note: The following is an edited version of a press release from Shire. Read the press release from Shire in it’s entirety here. Shire has announced the submission of an investigational new drug (IND) application to the U.S. Food and Drug Administration (FDA) for SHP654, also designated as BAX 888, an investigational factor VIII (FVIII) gene therapy for the […]

Industry News

New Biopharmaceutical Company, Sigilon Therapeutics, Launches

Note: The following is an edited version of a press release from Sigilon Therapeutics. Read the press release from Sigilon Therapeutics in it’s entirety here. Flagship Pioneering, a fully-integrated life science innovation enterprise, recently announced the launch of Sigilon Therapeutics Inc. Sigilon Therapeutics is a biopharmaceutical company that discovers and develops category-defining biocompatible encapsulated cell therapies. Sigilon Therapeutics’ discovery platform combines cell engineering and […]

Industry News

Genentech Announces Results in Emicizumab Phase III Studies (HAVEN 1 And HAVEN 2) in Hemophilia A With Inhibitors

Genentech, a member of the Roche Group announced today positive data from the primary analysis of the Phase III HAVEN 1 study in adults and adolescents and interim analysis of the Phase III HAVEN 2 study in children evaluating once-weekly subcutaneous emicizumab prophylaxis (preventative) for the treatment of hemophilia A with inhibitors to factor VIII. […]

Page 1 of 2412345678910NextLast »

Assisting and Advocating for the Bleeding Disorders Community