lenadogene nolparvovec

Lenadogene nolparvovec is an investigational gene therapy developed for the treatment of Leber hereditary optic neuropathy (LHON), a rare mitochondrial genetic disorder that causes rapid and severe vision loss, primarily in young adults. The drug uses an adeno-associated virus (AAV) vector to deliver a functional copy of the human ND4 mitochondrial gene (rAAV2/2_ND4) into retinal ganglion cells. This approach aims to restore the function of NADH dehydrogenase subunit 4, which is defective in LHON patients with the m.11778G>A mutation. Clinical trials have shown that a single intravitreal injection can lead to sustained improvements in visual acuity over several years, with bilateral injections offering greater benefit than unilateral treatment. The safety profile has been favorable, with mild intraocular inflammation as the most common adverse event[1][5][6][7].

Brand names

Lumevoq

Other names

lenadogene nolparvovec