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voretigene neparvovec · 1 trial · 1 indication
Full-field light sensitivity threshold (FST) is evaluated using white light, as averaged over both eyes.
| Arm | Type | Description |
|---|---|---|
| Voretigene neparvovec | EXPERIMENTAL | 1.5 E11 vg (0.3 mL subretinal injection in each eye, 6-18 days apart) |
| Name | Type | Description |
|---|---|---|
| voretigene neparvovec | GENETIC | Voretigene neparvovec is an adeno-associated viral type 2 (AAV2) gene therapy vector driving expression of normal human retinal pigment epithelium 65 kDa protein (hRPE65) gene. |
Inclusion Criteria: * Japanese participants with biallelic RPE65 mutation-associated retinal dystrophy; molecular diagnosis of RPE65 mutation must be confirmed by a Novartis designated laboratory in Japan. * Age four years or older. * Visual acuity worse than 20/60 (both eyes) and/or visual field l...
Voretigene neparvovec is a gene therapy developed by Novartis AG (ticker NVS) for biallelic RPE65 mutation-associated retinal dystrophy, an inherited eye condition. It is an investigational product that has been studied in a Phase 3 clinical trial in Japan. It is administered as a gene therapy in the ophthalmology therapeutic area.
Voretigene neparvovec is used for the treatment of biallelic RPE65 mutation-associated retinal dystrophy, a genetic retinal condition affecting the eye. The clinical program has focused on patients with this specific mutation, with biomarker-selected enrollment. The Phase 3 study enrolled patients aged 4 years and older in Japan.
Voretigene neparvovec targets RPE65, an exogenous gene. It is designed to deliver a functional copy of the RPE65 gene to address the genetic defect underlying biallelic RPE65 mutation-associated retinal dystrophy. The therapy is classified as a gene therapy in the ophthalmology therapeutic area.
Voretigene neparvovec is developed by Novartis AG, which trades under the ticker NVS. Novartis is the sponsor of the clinical development program for this gene therapy in biallelic RPE65 mutation-associated retinal dystrophy.
Voretigene neparvovec is in Phase 3 development. The Phase 3 trial, NCT04516369, has been completed. The program is investigational and remains in clinical development for biallelic RPE65 mutation-associated retinal dystrophy.
Voretigene neparvovec has been studied in one clinical trial, NCT04516369, titled Study of Efficacy and Safety of Voretigene Neparvovec in Japanese Patients With Biallelic RPE65 Mutation-associated Retinal Dystrophy. This Phase 3 trial was completed, enrolled 4 patients in Japan, and was an uncontrolled study without randomization or blinding.