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EPIC-241

Development stage: Discontinued
Lead developer: Epicrispr Biotechnologies
Modality: Gene Silencing → Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics, Gene Editing → Gene Therapies, Gene Addition/Replacement → Gene Therapies

Overview

EPIC-241 is a preclinical gene therapy program developed by Epicrispr Biotechnologies (Epic Bio) that uses the company’s non-cutting CRISPR-based epigenetic engineering platform to treat alpha-1 antitrypsin deficiency by modulating expression of the alpha-1 antitrypsin gene. Rather than editing DNA directly, Epic Bio’s compact CasMINI DNA-binding constructs are designed to be delivered in vivo (for example via adeno-associated virus vectors) to targeted genomic loci to upregulate or downregulate gene expression through epigenetic effectors, providing a potentially durable yet reversible “gene expression modulation” approach.[1][3][5]

Targets

SERPINA1 (Alpha-1-antitrypsin)

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