Gene therapy upstart launches with $50M and long-term commercial goals

Spark Therapeutics CEO Jeffrey Marrazzo

Usually, when an academic research group comes up with something new and promising in the field of therapeutic development, it's up to venture investors to pick up the tab for clinical-stage assets and biotech building. But after almost a decade-long effort funding cutting-edge gene therapy work at a research arm of The Children's Hospital of Philadelphia, hospital officials decided to fund the company-building effort themselves and hang on to much of the equity rather than settle for a slice of future revenues.

A group of gene therapy experts with a pivotal-stage therapy that's already posted promising early-stage data is being spun out of The Children's Hospital of Philadelphia. The startup biotech is launching with a $50 million funding commitment from the hospital to pursue their goal of becoming a "fully integrated" company with a full pipeline of projects as well as commercial products. And they plan to play a prominent role in the rebirth of gene therapy, a trend that has helped fuel the development of several new companies, including bluebird bio and GenSight.

Spark Therapeutics got underway a few months ago, says CEO Jeffrey Marrazzo, with a late-stage though small study for a gene therapy to fight inherited blindness caused by mutations in the RPE65 gene. There's also a small Phase I/II study in the clinic for hemophilia B.

Work on these gene therapies had begun at CHOP's Center for Cellular and Molecular Therapeutics, which launched 9 years ago. And now a group of the people who were involved in the work are taking positions at Spark with plans to build a full slate of clinical, regulatory and manufacturing expertise as some prominent investigators--including Jean Bennett--take roles as scientific advisers to the newly created biotech.

"The vision here is to build upon the fully integrated capabilities already in place" at CHOP, Marrazzo tells FierceBiotech, "and build a fully integrated gene therapy company that can cure as many diseases as possible and expand over time. That may eventually mean other capital partners or strategic partners."

Bennett was the co-author of a recent micro study that used gene therapy to treat a handful of patients with Leber congenital amaurosis, which leads to blindness. Bennett used an adeno-associated virus to transport a corrective gene into the eyes of three adult patients. Bennett said the patients responded well, demonstrating improved vision. And significantly, the treatment was completed without any harmful immune response, which has raised questions in the past about the safety of gene therapy.

The same technology was used to launch GenSight Biologics in Paris recently. The French biotech, backed by some big European funds, reported earlier this year that it is taking the same gene therapy approach for Leber's hereditary optic neuropathy, along with its optogenetic therapy for retinitis pigmentosa. The new Leber's project will use the same vector Bennett used to transfer a corrective mitochondrial gene into the cells.

It's typical in most academic organizations to look to out-license their best work, adds Marrazzo. From CHOP's perspective the "real expertise and talent all resided, here and not out there in the marketplace. Transferring out wouldn't give them the same share of the value pie."

The company only has a small startup team at work now, says the CEO, but he expects the staff to swell to "the dozens" within a year.

- here's the press release