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Spark Therapeutics Achieves Recruitment Goal in Phase 3 Gene Therapy Clinical Study for Inherited Blindness

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 faces and ambulate independently.  All school-aged patients enrolled in the trial were able to transfer from Braille classrooms to sighted classrooms.

In addition to Spark's Phase 3 recruitment achievement, Marrazzo shared several corporate updates at the J.P. Morgan conference, including: 

  • The company is well-positioned to be the first to obtain U.S. FDA approval for a gene therapy product and is preparing to launch the first in a series of therapies to address the significant unmet need of patients with inherited retinal dystrophies.
  • Spark expects to file an investigational new drug (IND) application for a second inherited retinal dystrophy this year.
  • The company presented encouraging results from the first subject in its Phase 1/2 hemophilia B program, in which a study participant has been able to go without factor for six months since vector infusion.
  • Two new team members have joined Spark: Carol Greve-Philips, senior vice president, business development and Joseph La Barge, senior vice president of business administration and general counsel. Greve-Philips previously served as vice president of corporate development at Genzyme; La Barge played a critical role as general counsel at Tengion, Inc., developing intellectual property strategies for first-in-class, cell-based therapies. 

The company continues to enroll its Phase 1/2 trial for hemophilia B and is advancing toward the clinic with gene therapy programs to address multiple, fatal neurodegenerative diseases.

For more information on Spark Therapeutics please visit or follow on Twitter @spark_tx.

About Spark Therapeutics
Spark Therapeutics is developing potentially curative, one-time gene therapy products to transform the lives of patients and re-imagine the treatment of debilitating diseases. Spark's lead gene therapy candidate, for RPE65-related blindness, is currently in Phase 3 clinical trials with the potential to be the first approved gene therapy in the U.S., and the first in a series of therapies to address the significant unmet needs of patients living with blindness due to inherited retinal dystrophies.

Spark's founding team includes scientists who led the movement to develop gene therapy as a new treatment paradigm, establishing clinical proof of concept in the eye and liver and contributing key insights to the field that have resulted in a resurgence of industry interest in gene-based medicines. In addition to the Phase 3 program in RPE65-related blindness, the company has a Phase 1/2 program in hemophilia B, and preclinical programs to address neurodegenerative diseases and other inherited retinal dystrophies and hematologic disorders. Spark has rights to a proprietary manufacturing platform that has an unparalleled track record of success in supporting clinical studies across diverse therapeutic areas and routes of administration. The company's expertise across research, clinical, regulatory and manufacturing builds on a legacy of innovation and excellence in gene therapy established by Spark's team while at The Children's Hospital of Philadelphia Center for Cellular and Molecular Therapeutics. To learn more visit

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