by Jo Kaur, Founder of Riaan Research Initiative
(June 17, 2026) - Last week, on June 9, 2026, we had the honor and privilege of announcing that Riaan became the first child in the world to receive a gene therapy for Cockayne syndrome.
In less than five years, Riaan Research Initiative, with the support of our donors, research partners, and collaborators, moved an entire drug development program from concept to clinic. In the biotech world, this is extraordinary speed. It was possible only because you believed in us.
We want to take a moment to send a heartfelt thank you to everyone who has supported this work in any way. You are part of this remarkable achievement.
Read our press release here.
View and share our announcement video above and here.
We are also grateful to our many partners, including the global law firm Hogan Lovells, which helped us secure IND clearance from the FDA.
Read Hogan Lovells’ press release here.
Earlier this week, we had the opportunity to sit down with Andelyn Biosciences, our manufacturer, for a thoughtful and candid interview about Riaan’s story, parent-led drug development, advice for manufacturers and others in the industry, and reflections for rare disease parent-foundations beginning the exciting but complex journey of building treatments for their children and communities.
Most importantly, we were able to highlight the joy Riaan brings to our family and to everyone who knows him, and to honor the positivity and fullness of life that every child in our community brings to this world.
Below are two excerpts from the interview. The full interview is available here on the Andelyn blog.
Question: For people meeting you and Riaan for the first time, what do you most want them to understand about your family’s journey?
Kaur: I want them to understand that this is a love story. I’ve never loved anyone as much as I love Riaan. That love propelled me into an unfamiliar research ecosystem, into conversations with scientists, clinicians, and manufacturers, with a confidence and authority I never could have imagined. When I was pregnant with Riaan, I met this kind, older woman in a candle shop at the Atlanta airport. We bonded, and she told me she always knew and understood babies, long before anyone else. Touching my belly, she said: “This one is special, this one is going to change the world.” I think she was right. Aside from the gene therapy, Riaan has brought tremendous, unbelievable joy to everyone, near and far. Our family has had the best six years together. We’re happy. I want people to understand that ours isn’t a story of suffering or tragedy. It’s a story of unconditional love and faith.
Question: What is your hope for the next family that may start a foundation?
Kaur: I know they’re desperate, and I understand that desperation. But I think every family starting should pause and try to understand the landscape before committing to a treatment path. Talk to as many foundations, academics, clinicians, manufacturers, and others in the field as they can. Take notes on these conversations, and trust their intuition. Build the right team from the very beginning and use funds strategically. Drug development can’t happen sequentially; they have to map out the entire chessboard from day one if they really want to get it right. Science matters, but scientific expertise does not always equate to mastery of regulatory or drug development. Choose collaborators who value urgency, humility, intellectual honesty, dedication to children living with rare diseases, and respect for everyone on the team. The right team will take families farther and faster than they imagined.
We know many families are asking what comes next. Our hope is to help make this treatment available to additional children, pending regulatory approvals and continued funding.
That next chapter will require the same urgency, generosity, and belief that brought us this far.
Please donate here to help us continue this work.
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