Technology Overview

Our SingleCutCRISPR gene editing has the potential to dramatically improve the lives of patients who suffer from neuromuscular diseases.

The technology underlying Exonics’ gene editing approach is licensed from the University of Texas Southwestern Medical Center and is based on research from the laboratory of Eric Olson, PhD, our scientific founder and chief science advisor. Gene editing is a revolutionary method for permanently correcting errors in the DNA sequence that cause the underlying disease.

Based on early preclinical results, Exonics’ SingleCutCRISPR gene editing technology has the potential to treat a large proportion of boys who suffer from Duchenne, by repairing mutations of dystrophin, the key protein missing in boys with Duchenne.

Exonics SingleCutCRISPR gene editing technology:

  • Uses cell’s natural mechanisms for gene repair to induce normal expression of dystrophin
  • Single cut makes minimum genomic modification, at precise location
  • Targets precise cell types to repair skeletal muscle, cardiac, diaphragm
  • Durable effect after one administration
  • Treats underlying cause of neuromuscular genetic diseases

gene editing

SingleCut CRISPR experiments conducted showed in a Duchenne mouse model a durable increased restoration in dystrophin production, as well as improved structure and function of both skeletal muscle and the heart, without any apparent side effects.