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 rare diseases where no, or only palliative therapies, exist.
Spark recently announced an exclusive option agreement with Clearside Biomedical to license Clearside’s microinjector technology for potentially differentiated delivery of gene therapies to the eye.