The Children’s Hospital of Philadelphia (CHOP), an institution on the forefront of gene therapy research for more than 20 years, has invested $50 million in Spark Therapeutics, a biotech start-up company based in Philadelphia. The company will take control of two clinical gene therapy trials that originated at CHOP: a phase III study for inherited blindness caused by mutations of the RPE65 gene and a phase I/II study for hemophilia B. The move marks a significant step in advancing the use of gene therapy to treat diseases by correcting or replacing dysfunctional genes.
Katherine A. High, MD, has spearheaded much of CHOP’s work in gene therapy for the last two decades. She is a Howard Hughes Medical Institute Investigator, the William H. Bennett Professor of Pediatrics at the University of Pennsylvania School of Medicine, and an attending physician at CHOP. High is also the director of CHOP’s Center for Cellular and Molecular Therapeutics (CCMT), established in 2004 as a leading center for gene therapy translational research and manufacturing. Many of CCMT’s leaders, including High, will take on management roles within Spark or act as scientific advisors.
“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 groundbreaking new treatments to patients in need.”
This partnership follows on the heels of a major breakthrough in gene therapy for hemophilia. In 2011, six hemophilia B patients at the University College London (UCL) Cancer Institute in London were successfully treated with gene therapy. The therapy incorporated the use of adeno-associated viruses (AAVs) as delivery vehicles, or vectors, to carry the genetic codes that trigger the production of the factor IX (FIX) protein, which is deficient in people with hemophilia B. Ideally, AAVs deliver the genetic material into living cells to sustain therapeutic effect without causing disease or triggering significant immune responses. The trial was expanded to eight patients, all of whom generated increased levels of FIX.
The AAVs for the study were prepared by a team from the St. Jude Children’s Research Hospital in Memphis, TN. The patients were recruited and treated with the therapy by investigators at UCL, while study co-author High and colleagues at CHOP have monitored patients for immune reactions.
According to a Spark news release, the company has entered into agreements with multiple academic institutions to assemble the technology, programs and capabilities needed to deliver its pioneering gene therapy products. Spark has exclusive rights to commercialize CHOP’s proprietary manufacturing technology and will use clinical-grade gene therapy vectors produced by the CCMT's state of the art good manufacturing practices clinical facility.
Spark Therapeutics develops gene-based medicines for a range of diseases. It has produced AAV vectors for clinical studies for the past 20 years. Its goal is to be the nation’s first commercial provider of gene therapy.
Source: The Philadelphia Inquirer and Spark Therapeutics news release, both dated October 22, 2013