A gene therapy spinout from Childrens Hospital of Philadelphia has added a head of regulatory affairs as it continues Phase 3 clinical trials for its treatment to reverse the effects of inherited retinal degeneration caused by a mutation in the RPE65 gene. It is the latest in a series of additions to the company following a $72.8 million Series B fundraise earlier this year. <\/p>\n
Daniel Takefman previously worked for eight years as chief of the gene therapy branch at the Center for Biologics Evaluation and Research at the U.S. Food and Drug Administration, according to a company statement. <\/p>\n
Spark Therapeutics, headed by CEO Jeffrey Marrazzo, has been bulking up its management muscle this year. Dr. Katherine High, the former founding director of the Center for Cellular and Molecular Therapeutics at CHOP and a pediatrician at the Perelman School of Medicine, became president and Chief Scientific Officer and has a board seat. It added Stephen Webster as CFO from Optimer Pharmaceuticals and Adolor Corp, whic Cubist Pharmaceuticals acquired in 2011. Steven Gelone joined as head of clinical research and development. Gelone had previously worked for ViroPharma in a similar role Shire acquired the company last year. Diane Blumenthal, another industry veteran, became head of technical operations. She had worked for Eli Lilly for 14 years. <\/p>\n
Romuald Corbau, former research project leader for Pfizers gene therapy program for the hepatitis C virus, joined as Sparks translational research lead to help support the companys pipeline of gene therapies. <\/p>\n
Earlier this month Spark received breakthrough therapy designation for its SPK-RPE65 treatment. The status speeds up the development and review of investigational therapies for serious or life-threatening conditions, particularly when theres an unmet need. <\/p>\n
Marrazzo said earlier this year that the company expects to have data from the Phase 3 trial available in the first half of 2015. In addition to its lead indication, Spark is also working on a Hemophilia B gene therapy treatment to reduce the doses of clotting factor that hemophiliacs need to take to avoid excessive bleeding. The condition is estimated to affect 20,000 people, according to the Centers for Disease Control. <\/p>\n<\/p>\n
[Photo credit: BigStock Photos] <\/p>\n
Get our daily newsletter or follow us. <\/p>\n
Please enter your email below: <\/p>\n
<\/p>\n
Read the original here:<\/p>\n
Spark Therapeutics adds regulatory insider to support gene therapy for retinal degeneration<\/a><\/p>\n","protected":false},"excerpt":{"rendered":" A gene therapy spinout from Childrens Hospital of Philadelphia has added a head of regulatory affairs as it continues Phase 3 clinical trials for its treatment to reverse the effects of inherited retinal degeneration caused by a mutation in the RPE65 gene. Continue reading