Drug intelligence / Profile preview

entacingene turiparvovec

Development stage
Phase 2
Lead developer
MeiraGTx Holdings
Modality
AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies
Administration
Intraocular, Subretinal, Ophthalmic
01

Overview

Entacingene turiparvovec is an investigational adeno-associated virus (AAV) based gene therapy designed to treat inherited color vision defects, specifically achromatopsia caused by mutations in the CNGB3 gene. The therapy uses an AAV serotype 8 vector to deliver a functional copy of the human CNGB3 gene directly to retinal cells via intraocular injection, aiming to restore or improve cone photoreceptor function and color vision. It is being developed by MeiraGTx in collaboration with Johnson & Johnson Innovative Medicine (formerly Janssen Pharmaceuticals), with originator rights from UCL Business. Entacingene turiparvovec has received orphan drug designation for color vision defects and is currently in Phase I/II clinical trials for achromatopsia[1][2][3][6].

Other names
Adenovirus associated viral vector serotype 8 containing the human CNGB3 gene
02

Targets

CNGB3 (Cyclic nucleotide-gated channel beta 3)

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