FDA clears IND application for trial of RPE65 retinal dystrophies treatment
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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.
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The multinational, multicenter, multiple-cohort, dose-finding study will evaluate the safety, tolerability, efficacy and long-term clinical durability of a single injection of HG004 in adult and pediatric participants for up to 52 weeks. After the study is complete, participants will be monitored in a long-term follow-up study. Trial initiation is expected in the first half of this year.
“We are thrilled to have received the IND clearance of our HG004 program from U.S. FDA, marking our first IND clearance as a company and our first retinal disorder program to reach clinical development stage,” Xuan Yao, PhD, co-founder and CEO of HuidaGene, said in the release. “The goal of the HG004 program is to develop a one-time, non-AAV2 gene replacement therapy to restore, treat and prevent blindness of children and adults with severe visual impairment or blindness due to RPE65 mutation-associated retinopathies globally.”
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