Genetic Engineering in the Press by GEG

Spark Therapeutics (NASDAQ:ONCE), a fully integrated gene therapy company dedicated to challenging the inevitability of genetic disease, and Pfizer (NYSE:PFE), today announced that, with a cumulative follow-up of more than 18 patient years of observation (5 to 121 weeks), all 15 participants in the ongoing Phase 1/2 clinical trial of investigational SPK-9001 for severe or moderately severe (FIX:C ≤ 2 percent) hemophilia B, had discontinued routine infusions of factor IX concentrates. None of the 15 participants experienced serious adverse events, and there were no thrombotic events or factor IX inhibitors, as of the May 7, 2018 data cutoff. These data will be presented today by Spencer K. Sullivan, M.D., hematologist and clinical investigator, Mississippi Center for Advanced Medicine, at the World Federation of Hemophilia (WFH) World Congress in Glasgow, Scotland, during the “Free Papers: Gene Therapy” session at 10:15 a.m. BST

Arie Belldegrun and David Chang steered Kite Pharma last year to a historic FDA approval and a $12 billion bear hug from Gilead Sciences. Now they’ve re-emerged with a new startup, Allogene, which has $300 million in backing and the rights to a CAR-T cell therapy program which is quite different from the one they brought to market with Kite.