Neurophth Biotechnology Ltd, a leading gene therapy specialist based in China, has announced the completion of patient enrollment and dosing in a Phase I/II clinical study for its investigational drug NR082 (rAAV2-ND4; NFS-01) in the United States. The study targets ND4-mediated Leber’s hereditary optic neuropathy (ND4-LHON).
The single-arm, multi-center Phase I/II study aims to assess the safety and efficacy of NR082 in treating ND4-LHON in the US. Initially approved for clinical trials in the US in January 2022, the drug received its first dosing in June 2023.
NR082 is a groundbreaking intraocular gene therapy candidate being developed to address Leber’s hereditary optic neuropathy (LHON) associated with the ND4 mutation. The drug’s mechanism of action involves the use of a recombinant adeno-associated virus as a carrier to deliver the correct gene to the damaged optic ganglion cells of patients via a single intravitreal injection. This approach is designed to repair the mitochondrial biological respiratory chain, thereby restoring the vitality and visual function of the optic ganglion cells.- Flcube.com
