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Rare Pediatric Disease Designation received by MeiraGTx

MeiraGTx Receives Rare Pediatric Disease Designation for the Treatment of LCA and Treats First Pediatric Patient in Phase 1/2 Clinical Trial

MeiraGTx, a London and New York-based gene therapy company, today announced that the Offices of Orphan Products Development and Pediatric Therapeutics of the U.S. Food and Drug Administration (FDA) have granted rare pediatric disease designation to the Company’s gene therapy product candidate AAV2/5-OPTIRPE65 (A001) for the treatment of patients with Leber’s Congenital Amaurosis due to mutations in the RPE65 gene (LCA2)