Drug intelligence / Profile preview

CG-511

Development stage
Preclinical
Lead developer
CureGene
Modality
Gene Addition/Replacement → Gene Therapies, Gene Editing → Gene Therapies, Gene Silencing → Gene Therapies
Administration
Ophthalmic
01

Overview

CG-511 is a preclinical-stage gene therapy being developed by CureGene for the treatment of achromatopsia, a rare genetic eye disorder. Achromatopsia is characterized by the loss of cone photoreceptor function, leading to total color blindness, severely reduced visual acuity, and light sensitivity. CG-511 utilizes an adeno-associated virus (AAV) vector to deliver a functional copy of a gene essential for cone cell activity (typically targeting mutations in the cyclic nucleotide-gated channel subunits such as CNGA3 or CNGB3) to the retina. By restoring the expression of these critical proteins, the therapy aims to improve visual function and color perception in affected patients. The program is currently undergoing preclinical evaluation to support future clinical development.

02

Targets

CNGA3 (Cyclic nucleotide-gated channel subunit alpha 3)

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