Healio

Opus Genetics completes dosing in first cohort of Leber congenital amaurosis trial

Please provide your email address to receive an email when new articles are posted on . The first cohort has finished dosing in the phase 1/2 clinical trial of OPGx-LCA5. The gene therapy, under ...
Healio

Positive early safety, efficacy data reported for Leber congenital amaurosis gene therapy

Please provide your email address to receive an email when new articles are posted on . No serious treatment-emergent adverse events were seen at 12 months. Patients who received a high dose of ...
Nasdaq

MeiraGTx Receives FDA Rare Pediatric Disease Designation for AAV8-RK-RetGC Program Targeting Leber Congenital Amaurosis

MeiraGTx receives FDA Rare Pediatric Disease Designation for AAV8-RK-RetGC to treat Leber congenital amaurosis due to GUCY2D mutations. MeiraGTx Holdings Plc announced that the FDA has granted its ...