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EDIT-102

Development stage
Preclinical
Lead developer
Editas Medicine
Modality
CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies, Gene Silencing → Gene Therapies, Gene Addition/Replacement → Gene Therapies
Administration
Ophthalmic
01

Overview

EDIT-102 is an experimental CRISPR-based genome editing medicine designed for the treatment of Usher Syndrome Type 2A (USH2A), a genetic condition characterized by hearing loss from birth and progressive vision loss due to retinitis pigmentosa. Developed by Editas Medicine, the therapy utilizes CRISPR/Cas9 technology to target and repair mutations in the USH2A gene, specifically aiming to restore the production of functional usherin protein in the retina. The program was originally part of a strategic alliance with Allergan (later acquired by AbbVie), but Editas Medicine regained full global rights to the asset in 2020. EDIT-102 is intended for subretinal administration to directly modify the genomic sequence in photoreceptor cells.

02

Targets

USH2A exon 13

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