Spark Therapeutics Presents Additional Phase 3 and Durability Data on SPK-RPE65 at Retina Society Annual Scientific Meeting

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Spark Therapeutics, Inc.
ONCE
announced today the presentation of additional data from the Phase 3 pivotal trial as well as continued durability data from an earlier Phase 1 study of SPK-RPE65, its lead product candidate for the treatment of RPE65-mediated inherited retinal dystrophies (IRDs).   Principal Investigator Stephen R. Russell, MD, of the Stephen A. Wynn Institute for Vision Research at the University of Iowa, presented Phase 3 data highlighting the rate, breadth and magnitude of changes seen across the primary endpoint and first secondary endpoint following administration of SPK-RPE65. In addition, Dr. Russell presented data on the three-year durability of improvements in the same measures of functional vision and light sensitivity in a cohort of subjects from an earlier Phase 1 trial. Today's presentation builds on top-line results of a randomized controlled multi-center Phase 3 trial announced by Spark on October 5, which demonstrated a highly statistically
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