A critical mission of our center is to bring novel therapies to the affected patients. We structured our center to facilitate translational research into clinical trials with speed and caution. We have collaborated on and assisted with developing multiple ocular gene therapy INDs, documents required by the FDA before initiating a clinical trial. In particular, our collaborative work on Leber’s Congenital Amaurosis type 2 (LCA caused by RPE65 mutations) with the Children’s Hospital of Philadelphia (CHOP) has resulted in the only Phase 3 gene therapy study currently underway in the world.
LCA2 is a rare form of inherited blindness that is symptomatic starting in infancy. In 2001, our team demonstrated the efficacy of gene therapy in a dog model of LCA21. Subsequently, we joined forces with CHOP, and brought the preclinical research into the clinical realm. Our team was the first to receive approval to conduct a gene therapy trial in pediatric subjects with a non-lethal disease. This paved the way for other groups to include children as well. The strategic alliance between our group and CHOP proved to be highly effective; the trial successfully and safely improved vision of most trial subjects2,3. The results were particularly impressive in the child participants who, before intervention, could not navigate independently or participate in normal classroom activities, but whose lives were transformed after intervention. We are currently working with Spark Therapeutics, LLC in the completion of the phase 3 study, and translating the program into a market product.
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