NFS-01

NFS-01 (also known as rAAV2-ND4) is an investigational gene therapy developed for the treatment of Leber hereditary optic neuropathy (LHON) associated with the G11778A mitochondrial DNA mutation. The therapy utilizes a recombinant adeno-associated virus type 2 (rAAV2) vector to deliver a functional, wild-type copy of the NADH dehydrogenase subunit 4 (ND4) gene directly to the retinal ganglion cells via a single intravitreal injection. By restoring the expression of the ND4 protein, the therapy aims to repair the mitochondrial respiratory chain complex I, thereby improving mitochondrial function and preventing the degeneration of the optic nerve. Originally developed at the Tongji Hospital of Huazhong University of Science and Technology, the program is being advanced clinically by Neurophth and has received IND clearance from both the China NMPA and the US FDA.

Other names

rAAV2-ND4-Huazhong University of Science and Technology-Leber hereditary optic neuropathyrAAV-2-ND4-Huazhong University of Science and Technology-Leber hereditary optic neuropathyrAAV 2-ND4-Huazhong University of Science and Technology-Leber hereditary optic neuropathy