lenadogene nolparvovec (Lumevoq)

Indications

• gene therapy for Leber hereditary optic neuropathy (LHON) with MT-ND4 gene mutation*

* not FDA-approved

Dosage

• intravitreal injection

Mechanism of action

• uses adeno-associated virus vector to deliver wild-type MT-ND4 gene directly to the mitochondrial membrane of retinal ganglion cells.

More general terms

• gene therapy
• ophthalmic agent (eyedrops)