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EPIC-141

Development stage: Preclinical
Lead developer: Epicrispr Biotechnologies
Modality: RNA Therapeutics → Nucleic Acid Therapeutics, Gene Editing → Gene Therapies

Overview

EPIC-141 is an investigational genetic therapy being developed by Epicrispr Biotechnologies for Retinitis Pigmentosa type 4 (RP4), a rare inherited retinal dystrophy. It is designed to modulate the RP4 gene using a CRISPR-based or related genome/RNA-editing platform to correct or counteract the pathogenic mutation driving photoreceptor degeneration, with the goal of preserving or restoring vision. The program is currently in the discovery stage and detailed information on its specific construct, delivery vector, and mechanism at the molecular level has not yet been publicly disclosed.

Targets

RHO (Rhodopsin)

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