Spark Therapeutics, a Philadelphia-based biotechnology company spun out of CHOP, is on their way towards creating the first gene therapy treatment for a genetic disease approved in the U.S.
The company has developed a drug to treat rare inherited blindness. The FDA accepted their application for a biologics license and granted it priority review. A test study of the drug, which streams genes directly to the eye’s retina, has shown that 97 percent of people treated experienced improvements.
“It’s really an exciting moment for medicine,” said Spark CEO Jeffrey D. Marrazzo in a Philadelphia Inquirer article.
Nothing like this drug currently exists in the U.S. The work that Spark is doing is one of the ways that Greater Philadelphia is pioneering health care and life sciences.