Clinical Trial Updates & News

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 12, 2017 – (Straits Times) – The use of stem cells to reverse liver cirrhosis - or the hardening of the liver - is being explored in a clinical trial. Conducted by a multi-centre team led by the National University Hospital (NUH), doctors aim to determine if stem cell therapy can improve liver function. 
July 12, 2017 – (Korea Herald) – South Korean regulators approved Wednesday the country’s first-ever cell-mediated gene therapy for degenerative arthritis Invossa, developed by US-based TissueGene, a wholly-owned subsidiary of Korea’s Kolon Group. Korea’s Ministry of Food and Drug Safety has granted approval to Invossa-K Inj., an injectable cell gene therapy for osteoarthritis, a joint disease common among the elderly in which the cartilage that cushions the ends of bones between joints breaks down. 
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 – (Spark) – Spark Therapeutics (NASDAQ:ONCE) announced that 10 participants in its ongoing Phase 1/2 clinical trial of SPK-9001 for hemophilia B, as of the June 5, 2017data cut off, had their AIR reduced approximately 99 percent to a mean of 1.0 annual infusion as of the data cut-off date, compared with 67.5 annual infusions before SPK-9001 administration.