Hospital launches gene therapy spinout with $50 million

The Children's Hospital of Philadelphia launched Spark Therapeutics LLC (Philadelphia, Pa.) with a $50 million capital commitment. The university granted Spark exclusive worldwide rights to develop and commercialize adeno-associated virus (AAV) vector-based therapies, including two clinical-stage programs: an AAV vector encoding the retinal pigment epithelium-specific protein 65kDa (RPE65) gene, which is in a Phase III trial to treat inherited blindness caused by RPE65 mutations; and an AAV vector encoding the factor IX gene, which is in a Phase I/II trial to treat hemophilia B. Data from the blindness trial are expected in 1H15. ...