Academia has long been an incubating source of incredible medical breakthroughs. Sometimes it takes a little push and risk-taking to take that progressive research out of the lab and into a business environment to achieve life-changing treatments for patients.
Such is the case for a promising new technology that has the potential to help 80% of boys with Duchenne muscular dystrophy. Duchenne is caused by a genetic mutation that impairs the muscle protein dystrophin. A permanent cure for Duchenne would include correcting the genetic mutation. Dr. Eric Olson at the University of Texas Southwestern Medical Center has been working on a gene editing technology called CRISPR/Cas9 that has the potential to correct many Duchenne mutations and diseases.
Dr. Olson is no stranger to advancing academic research beyond the lab, having co-founded multiple biotechnology companies. The companies, Myogen, Inc., miRagen Therapeutics, and Lone Star Heart, focused on therapies for heart muscle disease, cardiovascular disease, and heart regeneration and repair. With a $5 million commitment by CureDuchenne Ventures, Dr. Olson’s muscle repair research has become the foundation for a start-up company called Exonics Therapeutics with the purpose of rapidly accelerating this to clinic-ready stages.
This kind of groundbreaking technology takes time, even years, to be ready for patients. With our support, the development process is on a much faster track towards a promising outcome. Early studies in mice have already shown the repopulation of dystrophin after treatment, showing that we are headed in the right direction. If the CRISPR/Cas9 technology proves to be successful, the one-time treatment could potentially save the lives of many Duchenne patients and may be applicable to other genetic diseases.
By investing in programs and technological advancements such as CRISPR/Cas9, we ensure that any possibility for a cure is being explored. We will make sure that no stone will be left unturned on our path to discovering a cure to save the lives of the more than 300,000 boys affected worldwide.
CureDuchenne has committed $5M to accelerate Dr. Olson’s work, but we need everyone’s help to make sure we can deliver those funds immediately when needed so that his work is not slowed. CureDuchenne has set up a dedicated account so that donors may contribute directly to Dr. Olson’s specific CRISPR Cas9 project. Donate to help advance this research that could potentially correct a majority of the mutations in Duchenne. You can also share this flyer with those you know.