Ocular treatment likely to become first Gene Therapy approved in US

(c) nature.com

Spark Therapeutics has announced positive results from the Phase 3 pivotal trial of its lead gene therapy product candidate, SPK-RPE65, for the treatment of RPE65-mediated inherited retinal dystrophies (IRDs). These results represent the first successful randomized, controlled Phase 3 trial ever completed in gene therapy for a genetic disease.