Born of a Breakthrough

CHOP creates a for-profit company to advance gene therapy — and fund further research


The first decade of the 21st century was a time of remarkable progress at The Children’s Hospital of Philadelphia.

Researchers and clinicians had made incredible breakthroughs in vaccines, fetal surgery and cancer treatment — and countless other discoveries were on the horizon. But as the decade drew to a close, Hospital leaders became increasingly concerned about where funding for the next big breakthrough would come from.

So they decided to call on some truly extraordinary resources: their own.

A Bold Concept

In 2010, CHOP CEO Steven M. Altschuler, M.D., (above center) asked Jeffrey Marrazzo, M.B.A., M.P.A., (above right) a consultant and entrepreneur with vast experience in the healthcare industry, to identify projects already under way at CHOP that could be commercialized to benefit more patients and, in turn, return funds to the Hospital.

Marrazzo didn’t have to look far. Just a few months later, he came back to Altschuler with a promising candidate: the Center for Cellular and Molecular Therapeutics’ (CCMT) pioneering gene therapy work, which was already showing tremendous promise in treating a rare form of inherited blindness.

After much discussion with Board of Trustees members and Hospital administrators, Altschuler and his team decided that CHOP should create its own for-profit gene therapy company, with CHOP serving initially as its sole equity investor. The Hospital hopes someday to receive a return on this investment, which could then be used to fund additional research at CHOP.

In many ways, this is a logical next step in a long process. At a time when other institutions had turned away from gene therapy, CHOP had assembled a world-class team of gene therapy experts at the CCMT who were handling everything from manufacturing to clinical trials. The arrangement Altschuler and Marrazzo proposed would allow the CCMT team to remain deeply involved in the work and broaden its scope to benefit even more children.

“The gene therapy technology is so important to what we want to do as an institution,” says Altschuler. “I was looking for a way to move it forward that would allow us to further our research with the technology and provide us the best opportunity to integrate that with what we wanted to do clinically.”

“At CHOP, we do everything that a normal business would do to be successful, but at the end, we don’t give that money to shareholders. We reinvest that money in the community and to improve the health of children.”
— Steven M. Altschuler, M.D., CEO, The Children’s Hospital of Philadelphia

A Powerful Mission

The Hospital launched Spark Therapeutics in 2013, with Katherine High, M.D., (above left) former director of the CCMT, serving as one of the company’s scientific co-founders and as president and chief scientific officer, and Marrazzo in the role of co-founder and CEO. The blindness therapy, currently in a Phase III clinical trial, recently received breakthrough therapy designation from the U.S. Food and Drug Administration— and has the potential to be the first approved gene therapy for a genetic disease in the United States. “We’re in a good position to try to bring a product across the finish line and actually be able to help people who are born with a previously untreatable illness,” says High.

At the same time, the work has the potential to help many more children by generating funds to support new research. “It allows CHOP to invest in the next great idea,” says Marrazzo. “The money that was put into a place like the CCMT can be put into the next CCMT.”

And it goes on.

CHOP is a non-profit charity, and depends on donors like you to continue to provide What’s Next for the patients and families we serve.
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