Ron is a father of three and a medtech M&A lawyer and co-founder of the Yaya Foundation for 4H Leukodystrophy. Ron lives with his wife, June, and two kids, Ilan and Yasmin, in Minneapolis.
Give us the Yaya Foundation’s elevator pitch.
The Yaya Foundation is the only organization in the world fighting for kids and families affected by 4H Leukodystrophy, a rare and devastating genetic neurological disease that primarily affects children, results in significant disability and early death and that does not yet have a treatment or cure.
Through collaborative research, the Yaya Foundation is accelerating discovery of therapies and providing educational and emotional support to affected families.
In less than three years of existence, we’ve convened a global research network of patients, families, researchers, and clinicians collaborating in support of therapy discovery, launched an innovative data collection program, and been recognized by the Chan Zuckerberg Initiative as a best in class rare disease patient organization.
What led you to found the Yaya Foundation?
When my wife’s and my daughter, Yaya, was diagnosed with 4H when she was 8 months old, our geneticist told us there was nothing we could do.
No medicines. No therapies. No treatments. No cures.
Imagine being told that your child was sick and there was nothing you could do about it. Before Yaya’s diagnosis, I didn’t know that such a possibility could exist, and I didn’t accept it. I reached out to everyone I could – researchers, gene therapy and gene editing companies, rare disease venture capitalists, stem cell transplant experts. Sure enough, what our geneticist told us was true: there was nothing we could do to help our daughter fight her disease.
She died just 5 months later, and after losing her, my wife and I knew that we had to do whatever we could to ensure that no children like Yaya or families like ours would be told that no treatment alternatives existed that could help them. We founded the Yaya Foundation to ensure that in the future, kids like Yaya would have therapeutic alternatives available that would enable them to live better, longer lives.
What are the big milestones coming in the next few years for the Yaya Foundation?
We are relatively young as a patient organization and relatively early in our research journey as a disease community. In the coming years, we have a lot happening.
We just launched an exciting gene therapy proof of concept partnership with the Research Institute of the McGill University Health Center, the Montreal Children’s Foundation and the University of Massachusetts Medical School. This is the first step on the road to a gene therapy treatment for 4H and we hope to, over the next two years, demonstrate that gene therapy could be a viable therapeutic alternative for our patient community.
In parallel, we have several efforts focused on clinical trial readiness underway, including a data collection program in partnership with the Broad Institute of MIT and Harvard and a company called RARE-X, as well as a project focused on studying 4H natural history with labs in Amsterdam, Montreal, and Philadelphia.
We are also convening our patient, family, and research community in April 2022 in Montreal for our first ever 4H Leukodystrophy Family & Scientific Conference.
How do you think about engaging various strategic partners, including those within Medical Alley?
The Yaya Foundation seeks partners and donors who can help advance:
- Data collection and a natural history study that will support clinical trial readiness.
- Funding for high-risk, high-reward research studies that will enable our research community to generate early data that will unlock investment from more significant funding sources.
- Grants for early career researchers to attract new investigators to 4H.
- Funding for supportive programs for our community including a 4H Leukodystrophy Family & Scientific Conference.
- FInancial support for Foundation capacity building.
How do you balance leading an organization like the Yaya Foundation with your everyday life?
I have a relatively demanding day job and two little kids, and I often am only able to turn to Yaya Foundation work after my kids are asleep at night. My wife’s and my time with Yaya, and ultimately losing her, generated a lot of energy though, and the Yaya Foundation is my outlet for that energy. I view my work with the Foundation as an opportunity to continue to be Yaya’s dad, and I also feel a serious sense of responsibility to the kids and families all over the world that are affected by 4H and have put their hope and trust in the Yaya Foundation.
Maybe most importantly, I have the incredible luxury of getting to work with an amazing team at the Yaya Foundation and I know that I do not have to do this alone.
