Neurophth Approved to Start Gene Therapy for Hereditary Neuropathy

Neurophth Therapeutics, a Wuhan-San Diego rare disease company, was approved to start US trials of its in-vivo gene replacement therapy for Leber hereditary optic neuropathy (LHON) associated with ND4 mutation. The candidate, NR082, is a novel recombinant adeno-associated viral serotype 2 vector (rAAV2) containing a codon-optimized of NADH-dehydrogenase subunit 4 (ND4) gene. In three investigator-initiated trials that enrolled 186 LHON patients, an intravitreal injection of rAAV2-ND4 was well tolerated and showed efficacy in improving vision. NR082 is the first China-developed AAV gene therapy for LHON to start clinical trials. More details.... Share this with colleagues: // //