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CG-411

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

Overview

CG-411 is an investigational gene therapy candidate being developed by CureGene for the treatment of Leber congenital amaurosis (LCA). LCA is a group of rare inherited retinal dystrophies characterized by severe vision loss or blindness starting in early childhood. CG-411 is designed to deliver a functional copy of a gene to the retina, likely utilizing an adeno-associated virus (AAV) vector, to address the underlying genetic deficiency and potentially restore or preserve visual function. The program is currently in the preclinical stage of development as part of CureGene's ophthalmic gene therapy portfolio.

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