Crispr Halted Muscular Dystrophy In Dogs. Someday, It Might Cure Humans.

Dear Friends,

Today, our partner Dr. Eric Olson and his team at Exonics, a company CureDuchenne helped form, published data in the online journal, Science that shows an increase in dystrophin restoration of 92% in the heart of the canine’s. As the only Duchenne nonprofit organization to support Dr. Eric Olson’s work through Exonics, and to provide the seed funding necessary to accelerate his work in this company, we could not be more thrilled. This is a true testament to the power of venture philanthropy and I want to thank each and every donor and supporter who has believed in our work, and we are grateful to Dr. Olson for his dedication to Duchenne patients. Although we still have a long way to go before we confirm this therapy will be safe and effective in humans, this dramatic dystrophin restoration offers hope to all families affected by this disease.

The story began in 2015 when news of CRISPR technology first began surfacing. The CureDuchenne science and investment teams started to research and talk to different scientists. We met with a couple different groups before landing on Dr. Eric Olson, who had a long history of working in muscular disorders and had a true entrepreneurial spirit, which is crucial when working in this space.

After several meetings with UT Southwestern and Dr. Olson, we came to an agreement that the fastest way to accelerate CRISPR technology to human clinical trials was to form a company to fast track development. That’s when the three groups worked together in a truly collaborative partnership to form Exonics.

Within a matter of weeks, CureDuchenne committed $5 million in seed money to Exonics. We also put together an LLC to secure funding from other Duchenne organizations to help us to raise the $5 million, which we did not have, but every organization we talked to turned us down because they did not believe enough in the research project. Thankfully, after our first payment of $2 million, which was every penny we had, The Column Group came in with Series A funding and we no longer needed to fund the remaining $3 million.

With a mission to find, foster and fund investments in early stage projects, which many VCs and other investment groups avoid because of the high risk involved, this potential success story proves the incredible value in being a knowledgeable risk taker when it comes to finding cures for rare diseases.

On average, a drug takes 10 years to get to FDA approval after three years of clinic trials. With an average lifespan into the mid-20s, those with Duchenne need solutions faster. CureDuchenne Ventures’ goal is to identify the best science and to accelerate drug development timelines. We will continue to operate with the highest standards of ethics and integrity by re-investing the earnings back into research and programs that help support the whole journey of a Duchenne patient until we have a cure.

Read the full article HERE