CureDuchenne Ventures, a subsidiary of the nonprofit CureDuchenne that funds research to find a treatment for Duchenne muscular dystrophy (DMD), have committed $5 million in seed financing in Exonics Therapeutics, a new biotechnology company focused on utilizing gene editing technologies to advance the development of a DMD therapy.
DMD is an X-linked genetic progressive muscle disease affecting more than 300,000 boys worldwide. It is caused by a mutation in the gene encoding the protein dystrophin. Loss of dystrophin affects the stability of skeletal muscle, leading to progressive muscle damage and dysfunction. Children with the disease start missing development milestones at age 3 and often lose their ability to walk by age 12. All patients display reduced mobility, and ultimately respiratory or cardiac failure results in a reduced life expectancy and currently there is no cure for DMD.
Exonics’ CRISPR/Cas9 technology is a potential one-time treatment that would make a permanent correction of the mutation that causes DMD. The technology is licensed from the University of Texas Southwestern Medical Center and is based on the research of its scientific founder and chief science advisor, Dr Eric Olson. The initial seed funding will allow Exonics to advance the preclinical research findings of Dr Olson. Research in Dr Olson’s laboratory has demonstrated the potential of using adeno-associated virus (AAV) to deliver CRISPR/Cas9 to cells, to identify and correct exon mutations that prevent the production of dystrophin. Preclinical data published in Science suggests that this gene editing approach has the potential to permanently treat up to 80 percent of children who suffer from DMD.
Dr Olson, who also serves as Professor at the University of Texas Southwestern Medical Center commented: “This represents the next generation of potential Duchenne muscular dystrophy therapies. By leveraging the revolutionary CRISPR/Cas9 method to permanently correct errors in the DNA sequence, it is our hope that we can develop a one-time therapy that provides lifelong benefit to Duchenne patients.”
Source: Exonics to use CRISPR in an effort to treat majority of DMD boys. Press Release