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EPI-141

Development stage
Preclinical
Lead developer
Epicrispr Biotechnologies
Modality
CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies
Administration
Ophthalmic
01

Overview

EPI-141 is an investigational epigenetic gene therapy being developed by Epicrispr Biotechnologies (Epic Bio) for the treatment of Retinitis Pigmentosa 4 (RP4). RP4 is a hereditary form of retinal degeneration typically caused by mutations in the Rhodopsin (RHO) gene. EPI-141 utilizes Epic Bio's proprietary Gene Expression Modulation System (GEMS) platform, which employs a nuclease-deficient Cas9 (dCas9) protein fused to epigenetic modulators to precisely regulate gene expression at the transcriptional level without inducing double-strand DNA breaks. The therapy is delivered via an adeno-associated virus (AAV) vector and is designed to modulate the expression of the target gene to preserve photoreceptor function and prevent vision loss in patients with RP4.

02

Targets

RHO (Rhodopsin)

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