Therapeutics: Nuclear factor (erythroid-derived 2)-like 2 (NFE2L2; NRF2)

Mouse studies suggest NRF2 gene therapy could help treat retinitis pigmentosa (RP), optic nerve damage and other ophthalmic indications. In three mouse models of RP, subretinal injection of an adeno-associated virus (AAV) serotype 9 (AAV9) vector encoding NRF2 - a transcription factor that increases antioxidant gene expression - decreased morphological changes in cones and cone death and delayed loss in visual acuity compared with injection of a control vector. In a mouse model of optic nerve axonal injury, pretreatment with intravitreal injection of an AAV2 vector encoding NRF2 increased survival of retinal ganglion cells compared with control vector pretreatment. Next steps could include testing NRF2 gene therapy in models of other ophthalmic diseases involving oxidative damage.

Biogen Inc. and UCB group market the NRF2 pathway activator Tecfidera dimethyl fumarate to treat multiple sclerosis (MS). ...