Exonics Therapeutics, a Boston, Mass.-based biotechnology company, raised $5m in seed financing.
CureDuchenne Ventures, a subsidiary of the nonprofit CureDuchenne that funds research to find a cure for Duchenne muscular dystrophy, made the commitment.
The company intends to use the funds to advance the preclinical research of its scientific founder and chief science advisor Eric Olson, PhD, to a clinic-ready therapy.
Led by Dr. Jak Knowles, President and Interim Chief Executive Officer, Exonics Therapeutics focuses on utilizing gene editing technologies like CRISPR/Cas9 to advance the development of a treatment for Duchenne muscular dystrophy.
The company’s CRISPR/Cas9 technology is a potential one-time treatment that would make a permanent correction of the mutation that causes Duchenne.
Exonics’ laboratory has demonstrated the ability to use adeno-associated virus (AAV) to deliver a payload based on CRISPR/Cas9 technology that can identify and correct exon mutations that prevent the production of dystrophin, a protein that helps stabilize and protect muscle fibers.
The company’s and research activities are being conducted in Dallas, Tex.