Note: The below is an edited version of a press release by Spark Therapeutics. The original release can be read in it’s entirety here.
PHILADELPHIA, June 13, 2016 – Spark Therapeutics announced today updated results of the first cohort from the ongoing Phase 1/2 clinical trial of SPK-9001, the lead investigational candidate in its SPK-FIX program, which is being studied for the treatment of hemophilia B. The company also provided an update on preclinical data in its SPK-FVIII program for the treatment of hemophilia A.
“Spark’s validated platform has now successfully delivered gene therapies with proof of concept data in both the eye and the liver. We have made strong, tangible progress throughout our entire hemophilia franchise,” said Jeffrey D. Marrazzo, co-founder and chief executive officer of Spark. “The clinical data emerging from our hemophilia B program, which is partnered with Pfizer, and the strong parallels to the preclinical data from our rapidly progressing program in hemophilia A, give us early confidence in achieving our goal of eliminating the need for regular infusions to control and prevent bleeding episodes in patients with these diseases through a potentially one-time, intravenous administration of highly optimized gene therapies.”