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HuidaGene Doses First Patient in Gene Therapy Trial for Inherited Blindness

publication date: Nov 1, 2023

HuidaGene Therapeutics, a Shanghai-New Jersey gene editing company, has dosed the first patient in a China-US Phase I/IIa clinical trial for inherited blindness caused by RPE65 mutations. HuidaGene develops CRISPR-based programmable genomic medicines. HG004 is an adeno-associated virus 9 (AAV9) gene replacement therapy candidate aimed at inherited retinal dystrophies. The candidate uses a recombinant AAV9 vector to deliver a functional human RPE65 gene to the retina to restore, treat, and prevent blindness of children and adults with RPE65-IRDs. HG004 has been granted both orphan drug and rare pediatric disease designations in the US. More details.....

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