esonadogene mvoparvovec

Esonadogene mvoparvovec (Opvika, NR082) is an investigational gene therapy developed by Neurophth Therapeutics for the treatment of Leber hereditary optic neuropathy (LHON) caused by the G11778A mutation in the mitochondrial ND4 gene. The therapy utilizes a recombinant adeno-associated virus serotype 2 (rAAV2) vector to deliver a functional copy of the human wild-type ND4 gene directly to the retinal ganglion cells via intravitreal injection. By restoring the expression of the ND4 protein, a subunit of Complex I in the mitochondrial respiratory chain, the therapy aims to improve mitochondrial function, prevent further degeneration of the optic nerve, and restore visual acuity in affected patients. It has received Orphan Drug Designation from both the U.S. FDA and the EMA.

Brand names

Opvika

Other names

esonadogene imvoparvovecrAAV2-ND4rAAV-2-ND4rAAV 2-ND4