💊 Fármacos A-Z 🧬 Dianas 🛠 Herramientas 📚 Guías
Todos los fármacos Clases de fármacos Áreas terapéuticas Efectos Secundarios
Guías Glosario Línea de tiempo
Comparaciones Recorridos de fármacos Familias farmacológicas
🇺🇸 English 🇰🇷 한국어 🇯🇵 日本語 🇨🇳 中文简体 🇪🇸 Español 🇧🇷 Português 🇮🇳 हिन्दी 🇸🇦 العربية 🇫🇷 Français 🇷🇺 Русский 🇩🇪 Deutsch 🇹🇷 Türkçe 🇻🇳 Tiếng Việt 🇮🇩 Bahasa Indonesia 🇹🇭 ไทย

Voretigene Neparvovec

CHEMBL3707275 Phase 4 Aprobado Gene
Half-Life
—
Bioavailability
—
Protein Binding
—
Molecular Weight
— g/mol
LogP
—
Phase
4

Voretigene neparvovec is an adeno-associated virus serotype 2 (AAV2) gene therapy vector that delivers a functional copy of the RPE65 gene to retinal pigment epithelial cells in patients with biallelic RPE65 mutation-associated retinal dystrophy (a form of Leber congenital amaurosis or early-onset severe retinal dystrophy). RPE65 encodes the retinoid isomerohydrolase essential for the visual cycle, and its restoration improves light sensitivity and visual function in affected patients. It was the first directly administered gene therapy approved in the United States.

Mecanismo de acción

Delivers functional copies of a gene to target cells using a viral vector, restoring expression of the deficient protein and correcting the underlying genetic defect.

Pharmacokinetics (PK)

Pharmacodynamics (PD)

Mecanismo

Delivers functional copies of a gene to target cells using a viral vector, restoring expression of the deficient protein and correcting the underlying genetic defect.

HBD / HBA

- / -

No targets recorded

Target interaction data is not yet available for this drug.

No interactions recorded

Drug interaction data is not yet available for this compound.

No side effects recorded

Side effect data is not yet available for this drug.

Preguntas frecuentes

Voretigene neparvovec is an adeno-associated virus serotype 2 (AAV2) gene therapy vector that delivers a functional copy of the RPE65 gene to retinal pigment epithelial cells in patients with biallelic RPE65 mutation-associated retinal dystrophy (a form of Leber congenital amaurosis or early-onset severe retinal dystrophy). RPE65 encodes the retinoid isomerohydrolase essential for the visual cycle, and its restoration improves light sensitivity and visual function in affected patients. It was the first directly administered gene therapy approved in the United States.

Delivers functional copies of a gene to target cells using a viral vector, restoring expression of the deficient protein and correcting the underlying genetic defect.

Yes, Voretigene Neparvovec is an approved drug. It has reached clinical phase 4. It is classified as a Gene.

{# References & Data Sources section for drug detail pages. Renders standard pharmacological database links plus the drug's data_sources field. #}

References & Data Sources

  • ChEMBL — European Bioinformatics Institute (EBI). CHEMBL3707275. Open-access bioactivity database.

Data aggregated from publicly available pharmacological databases. Last updated 2026-02-27.

Aviso médico

This content is for educational and informational purposes only. It is not a substitute for professional medical advice, diagnosis, or treatment. Always consult a qualified healthcare provider before making medication decisions.

Data sources: ChEMBL, PubChem, DailyMed.