Collaboration to Fund CRISPR/Cas9 for Duchenne

Duchenne organizations are invited to fund CRISPR/Cas9 research at Exonics Therapeutics in a venture philanthropy collaboration.

We are making progress on treatments for Duchenne, but a cure is the goal our hearts long for. With the innovative CRISPR/Cas9 research being conducted by Dr. Eric Olson at the University of Texas Southwestern Medical Center, there is hope. CRISPR/Cas9 has the potential to correct as many as 80% of the many Duchenne mutations. This potentially could be a long-term cure by correcting the genetic mutation that causes Duchenne. CureDuchenne Ventures worked with Dr. Olson to form Exonics Therapeutics, a start-up company that may move this technology to drug development faster than a university lab. CureDuchenne Ventures also committed $5 million to Exonics to further the development of this research.

This research is costly, and as a Duchenne community we need to come together to support this effort.  An investment program, called a Special Purpose Vehicle (SPV), will allow individuals, foundations and other organizations to join in supporting Dr. Olson’s work. As Exonics reaches milestones and eventually clinical trials, there can be a financial return for those who invest using this Exonics/CRISPR SPV, allowing the organizations to use those proceeds to further fund their important mission.  The minimum investment is $100,000 and is limited to Accredited Investors.

CureDuchenne Ventures has already funded a significant amount of the SPV.  We invite Duchenne advocacy organizations to invest along-side. If you would like to contribute to Exonics/CRISPR SPV, there are several ways you can support this research:

• Donate to CureDuchenne specifically for this program: CureDuchenne.org/Exonics
• Encourage the Duchenne advocacy organization you work with to participate in this SPV
• Create a fundraising page or event to support this specific program
• Reach out to Accredited Investors you know and ask them to invest in this very important project.

CRISPR/Cas9 research is still in an early stage and we do not know if it will be as successful in humans as it has been in mice.  But, if successful, it has the potential to be life changing, not only for Duchenne families, but also for many diseases caused by genetic mutations. This kind of groundbreaking technology takes time, even years, to be ready for use by patients. In order to continue the development process on a steady pace, we need the support from individuals and organizations dedicated to finding a cure for Duchenne.

This is a wonderful opportunity for the Duchenne community to collaborate and possibly, together realize a financial reward as well as the real prize…a significant treatment for Duchenne. For more information on how to participate, please contact Debra Miller at debra@CureDuchenne.org or 949-872-2552. You can also donate directly at www.CureDuchenne.org/Exonics.