Voretigene Neparvovec

Mechanism :

Voretigene neparvovec-rzyl is an adeno-associated virus vector-based gene therapy that delivers a normal copy of the gene encoding human retinal pigment epithelial 65 kDa protein (RPE65) to retinal cells thus augmenting reduced or absent levels of biologically active RPE65. The RPE65 gene mutations lead to reduced or absent levels of RPE65 isomerohydrolase activity, blocking the visual cycle and ultimately impairing vision.

Indication :

• Treatment of confirmed biallelic RPE65 mutation-associated retinal dystrophy.

Contraindications :

There are no contraindications listed.

Dosing :

Adverse Effect :

Conjunctival hyperemia, cataract (including progression), increased intraocular pressure, retinal pigment epithelium tear, corneal thinning, eye disease (macular hole), retinal deposits, eye irritation, eye pain, maculopathy, ophthalmic inflammation, endophthalmitis.

Interaction :

There are no known significant interactions.

Hepatic Dose :