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HuidaGene Starts Multi-National Gene Therapy Trial in Patients With Inherited Form of Blindness

NEW YORK – HuidaGene Therapeutics on Wednesday said the first patient with a rare, inherited form of blindness has received its investigational gene therapy HG004 within a Phase I/IIa trial.

The Shanghai-headquartered firm is studying HG004's ability to restore, treat, and prevent inherited retinal dystrophies (IRDs) caused by mutations in the RPE65 gene, including Leber's congenital amaurosis (LCA), severe early childhood-onset retinal dystrophy, early-onset severe retinal dystrophy, and retinitis pigmentosa. Children with these conditions begin to experience night blindness starting at birth and up to age 5 and progressively lose their vision, which in turn affects their behavior, speech, and social development. There are 55,000 patients living in the world with RPE65-IRDs.

HG004 is designed to use a recombinant adeno-associated viral vector to deliver a functional RPE65 gene to the retina. In the recently launched multinational Phase I/IIa master protocol trial, researchers will assess the safety, tolerability, and efficacy of the gene therapy in patients with RPE65-IRDs. In addition to tracking patients' adverse events, lab results, and ophthalmic examinations, researchers will also evaluate patients' vision and ability to navigate a mobility course under different lighting conditions. After the initial study period, the company will also track patients' long-term outcomes after receiving the one-time HG004 treatment.

According to HuidaGene, HG004 is the first independently developed gene therapy that has orphan drug designation and rare pediatric disease designation from the US Food and Drug Administration. The same gene therapy product is also undergoing testing in China as a treatment for LCA type 2. In that nine-patient study, the last participant received HG004 in October. Adult and pediatric patients in that study have experienced visual improvements and there haven't been any safety issues with HG004, according to HuidaGene.

Following these promising findings in the LCA study in China, the company is hoping that this latest, larger Phase I/IIa trial can support regulatory approval and commercialization of HG004 in RPE65-IRDs. "We hope that this multiregional trial with the potential to be registrational globally can bring new, transformative therapeutic options for individuals living with RPE65-IRD worldwide," Xuan Yao, cofounder, president, and general manager for greater China at HuidaGene, said in a statement.