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

ces 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 www.sparktx.com or follow on Twitter @spark_tx.

About Spark Therapeutics
Spark Therapeutics is developing potentially curative, one-time gene therapy products to transfo
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