News from Our Members

July 13, 2017 – (Novartis) – "The panel's unanimous recommendation in favor of CTL019 moves us closer to potentially delivering the first-ever commercially approved CAR-T cell therapy to patients in need," said Bruno Strigini, CEO, Novartis Oncology. "We're very proud to be expanding new frontiers in cancer treatment by advancing immunocellular therapy for children and young adults with r/r B-cell ALL and other critically ill patients who have limited options. We look forward to working with the FDA as they complete their review." 
July 11, 2017 – (uniQure) – uniQure N.V. (NASDAQ:QURE) presented new clinical data demonstrating that the presence of pre-existing anti-AAV5 neutralizing antibodies does not predict the potential efficacy of AAV5-mediated gene transfer in patients with hemophilia B. 
July 11, 2017 – (BioMarin) – BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) announced today 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. 
July 10, 2017 – (Pluristem) – Austria’s regulatory health agency clears CLI study and joins the U.S., U.K., and Germany in conducting the 250-patient Phase III trial.  
July 10, 2017 – (uniQure) – The AAV5-based AMT-060 remains safe and well-tolerated with up to a year and a half of follow-up, with no serious adverse events and no development of inhibitors. All patients are now past one year of follow up with no loss of Factor IX (FIX) activity and no capsid-specific T-cell activation. 
July 10, 2017 – (CRISPR) – CRISPR Therapeutics (NASDAQ:CRSP) and Neon Therapeutics announced a research collaboration to explore the combination of each company’s proprietary technologies to develop novel T cell therapies. 
July 6, 2017 – (Shire) – SHP654 aims to protect hemophilia A patients against bleeds through the delivery of a long-term, constant level of factor expression.1The IND filing for SHP654 represents the latest step forward for Shire’s gene therapy program, which shows promise for both hemophilia A and B populations. 

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