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Neurophth Completes Dosing in Phase III Trial of Gene Therapy for Hereditary Blindness 

publication date: Feb 21, 2023

Suzhou Neurophth Biotech reported it has dosed the last patient in a China Phase III trial of its gene therapy for Leber hereditary optic neuropathy, which causes blindness in adult patients. The company said it hopes to market the product soon. NR082 is a novel recombinant adeno-associated viral vector, serotype 2, containing a mitochondria codon-optimized NADH-dehydrogenase subunit 4 (ND4) gene. In investigator-led studies, the ND4 gene therapy produced durable clinical responses of up to 90 months. The therapy is a one-time therapy for a disease that currently has no effective treatments. More details....

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