IND for Gene Replacement Therapy for Inherited Retinal Dystrophy Cleared by FDA

The company is developing a one-time, non-AAV2 gene replacement therapy to restore, treat, and prevent blindness of patients with RPE65 mutation-associated retinopathies.

This article was previously published on our sister site, Ophthalmology Times.

The FDA has approved an investigational new drug (IND) application for HuidaGene Therapeutics' clinical trial of HG004, a one-time, non-adeno-associated virus serotype 2 (AAV2) gene replacement therapy intended to treat children and adults with RPE65 mutation-associated inherited retinal dystrophies

According to the release, the planned multi-national, multicenter, multiple-cohort, dose-finding clinical trial will evaluate HG004 in adult and pediatric subjects with RPE65 retinopathies under a single master protocol in various countries.

The HG004 project aims to develop a single-injection, non-AAV2 gene replacement therapy using the same dose used in AAV2-based research, according to the company.

Head-to-head preclinical comparisons of HG004 and AAV2-based gene therapy at the same, single dose showed a recovery of retinal functions in mice by 67.6% with HG004 and 35.8% with AAV2 products at week 17 according to the release.

"We are excited by the promise of HG004 to offer a potential transformative treatment better than AAV2-mediated gene replacement therapy," said Hui Yang, PhD, co-founder and chief scientific advisor of HuidaGene. "Our extensive preclinical studies demonstrated superior transduction efficiency and substantial restoration of vision loss at the RPE layer when HG004 compared to AAV2 through our independently-developed RPE65 gene knockout murine disease model, which is found to mimic the retinal phenotypes and functions of patients with RPE65 mutation-associated inherited retinal dystrophies."

According to the company, patients enrolled in the investigator-initiated trial (IIT) in China at the end of 2022 that were progressing toward complete blindness saw a substantial restoration of vision with “nearly 25-fold lower vector doses” of HG004 than the approved AAV2-hRPE65 gene therapy product.

The company expects to initiate the trial in the first half of this year.

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