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.
Spark appoints 15-year FDA veteran Dr. Daniel Takefman, former Chief, Gene Therapy Branch within the Division of Cellular and Gene Therapies, Center for Biologics Evaluation and Research, as head of regulatory affairs. Dr. Takefman is recognized as a leader in the regulatory aspects of cell and gene therapies.