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miniTET-activator
- Development stage
- Preclinical
- Lead developer
- Epicrispr Biotechnologies
- Modality
- CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies, Fc-Fusion Proteins → Carrier/Scaffold Proteins → Recombinant Proteins and Enzymes
- Administration
- Intravenous
01
Overview
miniTET-activator is a hypercompact CRISPR-based epigenetic editor developed by Epicrispr Biotechnologies (Epic Bio) for the treatment of Friedreich’s Ataxia (FRDA). It is a component of the company's Gene Expression Modulation System (GEMS) platform. The therapeutic consists of a dCasONYX (a catalytically dead Cas variant) fused to a miniaturized demethylase from the TET family (miniTET) and a transcriptional activator. Delivered via adeno-associated virus (AAV), the system targets the silenced frataxin (FXN) gene, reversing repressive epigenetic marks—specifically DNA methylation and histone modifications—caused by GAA trinucleotide repeat expansions in intron 1. By reactivating the FXN locus, it restores frataxin protein to physiological levels, aiming to reach the therapeutic threshold required to treat the multisystem disorder without causing toxic overexpression.
02
Targets
Frataxin gene promoter (FXN promoter)FXN (Frataxin)
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