How did it all start?
We had a cute little boy. Life was perfect. Then he was diagnosed with Duchenne muscular dystrophy (a rare progressive neuromuscular disorder), and they said it was fatal. We started CureDuchenne a year after he was diagnosed.
Talk us through the past 20 years.
We’ve funded 17 research projects that have gotten to human clinical trials. I like to use the analogy of Mount Everest. The first 10 years were about trying to get to base camp, which is no small feat. But it’s a more gradual climb. We got there in 2014. Now the clouds have cleared, and we can see the summit.
What are the keys to your success?
We invest in early-stage science. We helped fund the first drug that ever got approved for Duchenne. It put our flag in the ground. Our venture philanthropy model really set us apart. Once we invest in a project, venture capitalists and pharma companies put a lot of credence into that. We’ve invested $19.2 million into research that has led to follow-on funding of $3 billion.
What’s next?
Taking our decades of research and moving these treatments forward even faster. We have hope that we now could be helping the first generation of long-term survivors. For “older” guys like my son (now 26), we need to move quickly. We may not be able to reverse the disease, but we can slow things down and give them a much better quality of life. For little guys, there’s great hope that we can bring curative treatments to them in the next few years. We want to save this generation.
