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.
We are a leader in the field of gene therapy seeking to transform the lives of patients suffering from debilitating genetic diseases by developing one-time, life-altering treatments. Our initial focus is on treating orphan diseases where no, or only palliative therapies, exist.
Experienced biopharmaceutical industry professionals, including former Optimer, Eli Lilly, 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.