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.
We are excited to announce that Rogério Vivaldi, M.D., M.B.A., former head of rare diseases at Genzyme, a Sanofi company, has joined Spark as our first chief commercial officer. Dr. Vivaldi joins as we prepare to complete our lead, Phase 3 clinical trial for SPK-RPE65 to address inherited retinal dystrophies caused by mutations in the RPE65 gene.