The FDA cleared an investigational new drug application from HuidaGene Therapeutics for a clinical trial of HG004 for the treatment of RPE65 mutation-associated inherited retinal dystrophies, according to a press release.
The application is supported by data from a preclinical comparison study that investigated the efficacy of HG004, a one-time ophthalmic injection, for the treatment of RPE65 retinopathies compared with adeno-associated virus serotype 2 at the same dose.
The multinational, multicenter, multiple-cohort, dose-finding study will evaluate the safety, tolerability, efficacy and