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 initiates a Phase 1/2 clinical trial of SPK-CHM, its product candidate for the potential treatment of patients with choroideremia (CHM), a rare form of night blindness, expanding the company’s pipeline of potential treatments for rare, blinding conditions.