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Spark Therapeutics Achieves Recruitment Goal in Phase 3 Gene Therapy Clinical Study for Inherited Blindness
Date:1/14/2014

SAN FRANCISCO, Jan. 14, 2014 /PRNewswire/ -- Spark Therapeutics, a late-stage gene therapy company, has reached its patient recruitment goal for its most advanced clinical program, a Phase 3 study for inherited retinal dystrophies caused by mutations in the RPE65 gene. Spark's CEO, Jeffrey D. Marrazzo, is presenting this and other corporate updates today, at the 32nd Annual J.P. Morgan Healthcare Conference in San Francisco. Marrazzo said Spark was able to achieve its recruitment goal a year after the study's initiation and anticipates filing a BLA on the final data in 2015. Currently, there are no pharmacologic treatments for this or any form of inherited retinal dystrophy, which ultimately causes irreversible blindness.

"The fact that we were able to enroll patients in our lead clinical program so quickly and seamlessly reflects the critical, unmet medical need associated with inherited retinal dystrophies as well as the deep expertise of our founders and the hard work of the clinical teams at The Children's Hospital of Philadelphia and the University of Iowa," said Jeffrey D. Marrazzo, co-founder, president and chief executive officer. "In order to realize our vision of transforming the lives of patients in a single therapeutic dose, we must continue to operate at the highest standard, as we have in achieving patient recruitment for Phase 3 of our lead candidate right on the heels of our corporate launch."

Spark's patient recruitment goal for the Phase 3 study was a minimum of 24 patients. The open-label, randomized, controlled study builds on a Phase 1/2 clinical trial in which 12 patients with RPE65-related blindness demonstrated notable improvements in functional vision, moving in some cases from being profoundly blind to being able to recognize fa
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