Exonics Therapeutics develops SingleCut CRISPR technology to repair mutations that cause Duchenne muscular dystrophy and other neuromuscular diseases. The company is advancing a lead gene‑editing program in Duchenne and aims to create a one‑time treatment that could provide lifelong benefit. Published preclinical data in mice suggest the approach can identify and repair exon mutations that prevent production of dystrophin. Exonics is generating additional data to support development of a safe and efficacious therapy. The SingleCut technology was licensed from the University of Texas Southwestern Medical Center and is based on research from Eric Olson’s laboratory. The company is led by founder and chief science advisor Eric Olson and CEO John Ripple. Exonics Therapeutics is advancing CRISPR/Cas9-based, one-time gene-editing therapies intended to permanently correct exon mutations that prevent dystrophin production in Duchenne muscular dystrophy. Its approach uses adeno-associated virus (AAV) to deliver a CRISPR/Cas9 payload that can identify and correct exon mutations; published preclinical data suggest the approach could potentially treat up to 80% of affected children. The company’s technology is licensed from the University of Texas Southwestern Medical Center and is based on the research of scientific founder and chief science advisor Eric Olson, PhD. Exonics will use the seed financing to advance Olson’s preclinical work toward a clinic-ready therapy. The company’s corporate office is in Boston, and its research activities are being conducted in Dallas. CureDuchenne Ventures and CureDuchenne’s relationships with the Duchenne community are positioned to support acceleration of development.

Total raised

$45M

Funding rounds

2

Latest round

Series A

Latest activity

Nov 2017