Yannick, 13, was blind before receiving gene therapy, which restored much of his sight.
Dr. Katherine High has pioneered the development of gene therapy for two decades.
Gene therapy is giving Michael, 67, new hope for a life free from hemophilia B.
Spark to open permanent west Philadelphia headquarters.
The team at Spark Therapeutics has pioneered the development of gene therapy for nearly two decades. Our founding team has brought us closer to solving the most complex challenges facing the field, creating new opportunities for the patients we serve and for the industry at large. Learn how we’re working to spark the next wave in the gene therapy revolution.
Experienced biopharmaceutical industry professionals, including former Optimer, ViroPharma, Shire and Pfizer leaders, recently joined Spark’s leadership team. Their expertise has enhanced Spark’s financial, clinical, operations and research capabilities as it prepares to complete development of SPK-RPE65, our lead gene therapy program for the treatment of inherited retinal dystrophies caused by mutations to the RPE65 gene. These rare blinding diseases result in severe visual impairments, progressing ultimately to blindness.