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HuidaGene’s CRISPR Therapy Approved to Start US Trial for Inherited Retinal Disease

publication date: Jan 30, 2023

HuidaGene Therapeutics, a Shanghai-New Jersey gene editing company, was cleared to start a US Phase I trial of its lead product, a CRISPR-based genomic medicine for patients with inherited RPE65 retinal dystrophies. RPE65 retinal dystrophies are a group of genetic diseases caused by mutations in the RPE65 gene that affect the retina, causing blindness in 100% of patients by age 40. The company believes its optimized CRSPR/Cas-based RNA editing tools are smaller, more efficient, and more specific than the competition, allowing lower dosing of its AAV vectors. It expects the candidate will be a one-time therapy for RPE65 retinal disorders. More details....

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