HuidaGene Therapeutics has received clearance from the US Food and Drug Administration (FDA) for its investigational new drug (IND) application submitted for a clinical trial of HG004 to treat RPE65 mutation-associated inherited retinal dystrophies in patients.
The planned multi-national, multi-centre, multiple-cohort, dose-finding clinical trial will evaluate HG004 in adult and paediatric subjects with RPE65 retinopathies under a single master protocol in various countries.
It aims to assess the tolerability, safety, efficacy, and long-term durability of HG004 when given as a single injection for up to 52 weeks.
Certain laboratory measures, adverse events, and ophthalmic examinations are the primary endpoints of the trial.
Additionally, the trial will evaluate visual function through a multiluminance mobility test (MLMT).
In this test, subjects will navigate a mobility course under different light levels.
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They will continue to be evaluated in HG004’s long-term follow-up study after completion of the primary study period.
HuidaGene Therapeutics co-founder and CEO Xuan Yao said: “We are thrilled to have received the IND clearance of our HG004 programme from US FDA, marking our first IND clearance as a company and our first retinal disorder programme to reach clinical development stage.
“Clearance of this IND is a testament to the in-house pipeline development capabilities and high-quality preclinical data supporting HG004, as well as the strong CMC and analytics capabilities through our partnership with WuXi Advanced Therapies.”
The company expects to initiate the trial in the first half of this year.
Investigational HG004 is being developed for the treatment of RPE65 retinopathies.
Retinal dystrophies, a group of genetic diseases, occur when mutations in the RPE65 gene affect the retina.