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's 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 potential one-time, life-altering treatments. Our initial focus is on treating rare diseases where no, or only palliative therapies, exist.
Spark announces successful pivotal Phase 3 trial of SPK-RPE65 for rare, inherited blinding conditions.