China-based gene therapy specialist Neurophth Biotechnology Ltd has announced that the first patient has been dosed in a Phase III clinical study for its NR082 (rAAV2-ND4, NFS-01). The study is being conducted in China to evaluate the efficacy and safety of NR082, a recombinant adeno-associated virus serotype 2 (rAAV2), in treating ND4-mediated Leber’s hereditary optic neuropathy (ND4-LHON).<\/p>\n\n\n\n
Understanding Leber’s Hereditary Optic Neuropathy (LHON)<\/strong>
Leber’s hereditary optic neuropathy (LHON) is a maternally inherited mitochondrial disease with an incidence rate of approximately 1 in 31,000 to 1 in 54,000. Up to 90% of LHON cases are caused by three mitochondrial DNA mutations, with the G11778A mutation in ND4 being the most common, accounting for about 50% to 80% of cases. Currently, there is no effective treatment available for LHON.<\/p>\n\n\n\n