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Neurophth Starts China Trial of Gene Therapy for Hereditary Blindness

publication date: Jun 28, 2021

Neurophth Biotech, a Wuhan-San Diego gene therapy company, dosed the first patient in a China trial of a therapy for Leber hereditary optic neuropathy (LHON), which causes blindness in adult patients. The Phase I/II/III trial will evaluate the safety, tolerability and efficacy of a recombinant adeno-associated virus vector 2 carrying the NADH-dehydrogenase subunit 4 (ND4) gene. The disease currently has no cure. The safety and efficacy of ND4 gene therapy have been shown in three investigator-initiated trials with clinical durability of up to 90 months. More details....

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