The US Food and Drug Administration (FDA) has granted HuidaGene Therapeutics a rare pediatric disease designation (RPDD) to accelerate the development and review of its gene therapy candidate, HG004. This one-time gene replacement drug aims to combat inherited retinal diseases (IRDs) caused by mutations in the RPE65 gene, predominantly impacting children. Conditions include Leber’s congenital amaurosis and retinitis pigmentosa, which both cause significant vision loss. HuangGene’s candidate has shown superior recovery of retinal function than other similar treatments in preclinical studies. This FDA decision offers HuidaGene various benefits, including tax credits, application fee exemptions, and seven years of market exclusivity. HuidaGene Therapeutics expects to commence a trial investigating HG004 in September 2023.
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[Source: Pharmaceutical Technology, August 7th, 2023]