Spark Therapeutics, a new, fully integrated company developing gene-based medicines for a wide range of debilitating diseases, has launched with a $50 million capital commitment from The Children's Hospital of Philadelphia (CHOP) to advance and commercialize multiple ongoing programs with clinical proof of concept.
Spark will assume control over two gene therapy clinical trials that are currently enrolling patients: a phase III study for inherited blindness caused by mutations of the RPE65 gene and a phase I/II study for hemophilia B. The company also is advancing toward the clinic with gene therapy programs to address neurodegenerative diseases and additional hematologic disorders and forms of inherited blindness.
"The creation of Spark is the culmination of a decade-long commitment by CHOP and our founding team to drive the field of gene therapy forward during a time when many in the industry had moved away," said Jeffrey D. Marrazzo, president and chief executive officer of Spark Therapeutics. "Their vision and long-term dedication have enabled us to effectively address many of the key challenges facing the field and to emerge with one of the industry's most robust clinical-stage gene therapy pipelines; as well as exclusive rights to commercialize a proprietary manufacturing platform, supply from a world-class manufacturing facility and a founding team with a proven track record of executing safe and effective gene therapy trials for nearly two decades. We are working with great urgency and care to deliver gene therapy products with the potential to transform the lives of those affected by severe genetic diseases."
Spark builds on the work of CHOP's Center for Cellular and Molecular Therapeutics (CCMT), established in 2004 as a center for gene therapy translational research and manufacturing. Many of the CCMT's leaders will assume management roles within Spark or engage with the company as scientific advisors, including Katherine A. High, M.D, a gene therapy pioneer who has been director of the CCMT since its inception.
"Gene-based medicines are among the most complex therapeutics ever developed," said High. "We at CCMT have persevered through more than a decade of scientific and clinical development and are now closer than ever to realizing the ambitious vision of one-time, potentially curative therapies to address serious genetic conditions. The team at Spark has incredible goals for the treatment of diseases including hemophilia B and inherited blindness, and we look forward to working with them to deliver new treatments to patients in need."