Founded in March 2013 as a result of the technology and know-how accumulated over two decades at the Children’s Hospital of Philadelphia (CHOP), Spark Therapeutics is a publicly traded, late clinical-stage gene therapy company. Its investigational therapies have the potential to provide long-lasting effects, dramatically and positively changing the lives of patients with conditions where no, or only palliative, therapies exist. Greater understanding of the human genome and genetic abnormalities have allowed our scientists to tailor investigational therapies to patients suffering from very specific genetic diseases. This approach holds great promise in developing effective treatments to a host of inherited diseases. Our initial focus is on treating orphan diseases.