Tom Avril, Inquirer Staff Writer Posted: Wednesday, October 23, 2013, 2:01 AM

Children's Hospital of Philadelphia has invested $50 million in a new biotech start-up that seeks to be the nation's first commercial provider of gene therapy, company officials announced Tuesday.

Spark Therapeutics will assume control over two clinical trials that originated at the prominent teaching hospital - one in which patients with a rare form of blindness already have regained some vision, the other an early-stage effort to treat hemophilia B.

Jeffrey D. Marrazzo, Spark's CEO, said the goal was to tackle still more genetic diseases in the future, including other rare forms of blindness, blood disorders, and two neurodegenerative diseases that he declined to identify.

Children's Hospital has spun off companies before with the involvement of other investors, but this marks the hospital's first foray as a primary source of start-up funds, said hospital CEO Steven M. Altschuler.

The move marks a coming-of-age moment for gene therapy - the concept of treating disease by replacing or correcting faulty genes. Confined to the realm of research for decades, it now appears headed to the clinic on multiple fronts.

A gene-therapy treatment called Glybera has been approved in Europe for treatment of lipoprotein lipase deficiency, an affliction marked by increased levels of fat in the blood. And earlier this year, a Cambridge, Mass.-based gene-therapy company called bluebird bio Inc. raised more than $100 million in an initial public offering.

Gary J. Kurtzman, managing director for health care at Safeguard Scientifics, the Wayne-based technology and health-care investment company, said Spark's prospects looked promising. He cited the involvement of Children's Hospital researchers such as Katherine A. High, a primary force in the hemophilia and blindness trials and now one of Spark's scientific advisers.

"It's a bold move," Kurtzman said of the hospital's investment. "Based on the technology and the assets and the expertise that Dr. High and other people there have, I think it's a . . . very smart move."

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Children's Hospital of Phila. funds gene-therapy company

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