hemophilia B

Sanofi SA (NASDAQ: SNY) presented data from two Phase 3 studies of fitusiran for the prophylactic treatment with hemophilia A or B, with or without inhibitors. Data were shared at the American Society of Hematology (ASH) Annual Meeting.  Fitusiran, an RNAi drug designed to...

CSL Behring and uniQure N.V.'s (NASDAQ: QURE) etranacogene dezaparvovec, a hemophilia B gene therapy, achieved the pre-specified primary endpoint of non-inferiority in annualized bleeding rate (ABR) compared to baseline Factor IX (FIX) prophylactic treatment. The study...

Centessa Pharmaceuticals plc (NASDAQ: CNTA) has announced positive topline results from the Phase 2a part of AP-0101, the six-month repeat dose portion of the study evaluating SerpinPC in severe hemophilia A and B patients. SerpinPC was well-tolerated. As previously disclosed, one...

UniQure N.V. (NASDAQ: QURE) has announced 52-week data from its Phase 3 HOPE-B gene therapy trial of etranacogene dezaparvovec to treat hemophilia B.  Data demonstrated durable, sustained increases in Factor IX (FIX) activity at 52-weeks post-infusion with a mean FIX activity...

The FDA has removed the clinical hold on uniQure NV's (NASDAQ: QURE) hemophilia B gene therapy program after determining the company had satisfactorily addressed all identified issues related to a single patient diagnosed with hepatocellular carcinoma (HCC) in the HOPE...

Just two months after Centessa Pharmaceuticals launched after the merger of 10 private biotech companies, the company has filed a $100 million IPO for listing on NASDAQ. Founded by Medicxi, Centessa in February raised a $250 million Series A round of funding. The Centessa portfolio...

Six companies are set to go public on Friday. Below are details on the most anticipated biotech deal of the week. The two best deals of the week are thought to be Spark Therapeutics Inc (NASDAQ: ONCE) and Shake Shack (NYSE: SHAK). Spark Therapeutics is a gene therapy company focused on...