A novel gene therapy medication candidate called NR082 has been identified by Neurophth Therapeutics (rAAV2-ND4).
The medicine has received a Breakthrough Therapy Designation (BTD) from China's National Medical Products Administration's Center for Drug Evaluation (CDE).
Leber hereditary optic neuropathy (LHON) linked to mtND4 mutations is being treated with the new gene therapy product, NR082.
The gene product is a recombinant serotype 2 adeno-associated viral vector carrying a human ND4 codon-optimized gene that is under the promoter and enhancer of a cytomegalovirus.
Phase I, II, and III clinical trials are now being conducted following the China NMPA's approval of the IND.
To repair the mitochondrial respiratory chain, enhance the sensory function of the retinal ganglion cell (RGC), and restore visual function in LHON patients, the correction genes are delivered to the damaged RGC via the viral vector through intravitreal injection.