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EPI-331

Development stage: Preclinical
Lead developer: Epicrispr Biotechnologies
Modality: CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies, Classical Binding Small Molecules → Small Molecules, Gene Silencing → Gene Therapies, Covalent Small Molecules → Small Molecules, Gene Addition/Replacement → Gene Therapies
Administration: Intravenous

Overview

EPI-331 is an investigational adeno-associated virus (AAV)-delivered epigenetic gene therapy being developed by Epicrispr Biotechnologies (Epic Bio) for the treatment of Duchenne muscular dystrophy (DMD). Utilizing the company's proprietary Gene Expression Modulation System (GEMS) platform, EPI-331 is designed to upregulate the expression of utrophin (UTRN), a functional analog of dystrophin. In DMD patients, the absence of dystrophin leads to progressive muscle degeneration; by epigenetically activating the UTRN gene, EPI-331 aims to compensate for the lack of dystrophin and stabilize muscle fibers. The therapy employs a nuclease-dead Cas9 (dCas9) fused to transcriptional activators to specifically target the UTRN promoter without cutting the DNA. EPI-331 is currently in the preclinical stage of development.

Targets

DNM1L (Dynamin-related protein 1)

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