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Spark Therapeutics, Pfizer announce data SPK-9001 ongoing Phase 1/2 trial.

Spark Therapeutics (ONCE) and Pfizer (PFE) announced that, with a cumulative follow-up of more than 18 patient years of observation, all 15 participants in the ongoing Phase 1/2 clinical trial of investigational SPK-9001 for severe or moderately severe 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, or WFH, World Congress in Glasgow, Scotland, during the "Free Papers: Gene Therapy" session at 10:15 a.m. BST. "We are pleased to see all 15 participants, notably including the first four participants who have been followed for more than two years, continue to show that a single administration of SPK-9001 has resulted in dramatic reductions in bleeding and factor IX infusions, with no serious adverse events," said Katherine A. High, M.D., president and head of research & development at Spark Therapeutics. "Our commitment to gene therapy research across our hemophilia programs remains steadfast with the goal of developing a novel therapeutic approach with a positive benefit-risk profile that aims to free patients of the need for regular infusions, while eliminating spontaneous bleeding."

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Publication:The Fly
Date:May 22, 2018
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