MeiraGTx (NASDAQ:MGTX) has announced that its AAV8-RK-RetGC program for the treatment of patients with Leber congenital amaurosis due to GUCY2D mutations (LCA1) has been granted Rare Pediatric Disease Designation (RPDD)...
MeiraGTx (NASDAQ:MGTX) reported that AAV-RPE65, its investigational gene therapy for the treatment of retinal dystrophy, met the primary endpoint of safety and tolerability in a Phase 1/2 study, as well as demonstrated...