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  1. Blood, Vaccines and Other Biologics

2017 Meeting Materials, Cellular, Tissue and Gene Therapies Advisory Committee

2017 Meeting Materials, Cellular, Tissue and Gene Therapies Advisory Committee

October 12, 2017

On October 12, 2017, the Cellular, Tissue and Gene Therapies Advisory Committee (CTGTAC) will meet in an open session to discuss and make recommendations on the safety and effectiveness of Biologics License Application (BLA) 125610, voretigene neparvovec, submitted by Spark Therapeutics, Inc. The proposed indication (use) for this product is treatment of patients with vision loss due to confirmed biallelic RPE65 mutation-associated retinal dystrophy.