We’re Back from Boston
2026 ASGCT Annual Meeting
Our Takeaways from ASGCT 2026
Walking away from the American Society of Gene + Cell Therapy (ASGCT) annual meeting, one word keeps coming to mind: gratitude.
Gratitude for the brilliant scientists working every single day toward a cure for PURA Syndrome. Gratitude for our friends, family, and our incredible "big kids," Sid and Charlotte, who cheer us on even when it means missed bedtimes and a night or two apart.
The Tide is Turning
If there was one overarching feeling from this year's conference, it was momentum — and we felt it everywhere.
One year ago, we attended ASGCT for the first time. There was a single poster on PURA Syndrome. This year? Five. Five posters, a buzz in the hallways, and a palpable sense that the scientific community is paying attention. That's the difference a year of relentless advocacy makes.
Parent-led advocacy is changing the game. Groups like ours — and others like Finding Hope for Frizzle (FRRS1L) — are no longer waiting on the sidelines. We are demanding a seat at the table, driving the research, and owning the data. That shift is real.
One of the most exciting moments came from Dr. Kiran Musunuru's keynote. You may have seen his name in the New York Times — he's the physician behind the treatment of "Baby KJ," the first person ever to receive a personalized gene therapy. Dr. Musunuru described working with the FDA to create an "umbrella" clinical trial: one application covering multiple genetic variants, with the ability to add new ones in real time. The goal is to move faster to patients. His words: "Make a single clinical batch and get the patient treated." He also championed the concept of "genetic surgery" and envisioned a future with trained interventional geneticists — a more agile, accreditation-driven path that doesn't rely solely on the traditional FDA process.
Rare diseases are having a moment. But attention means nothing without approved treatments.
The People Who Inspire Us Most
The most moving moments weren't on the main stage. They were in the hallways — conversations with parent advocates on the same journey, including families who have suffered unimaginable loss and are still showing up and fighting.
One line from advocate Chrissy Green, speaking on a panel with Viralgen, has stayed with us: "Find partners who value what you value."
That is the standard we hold ourselves to — from The Jackson Laboratory (JAX) to Dr. Jiang's lab at Yale University. Transparency and data sharing are non-negotiable for us, because the mission is too important for anything less.
This year fellow PURA parents Celena Lozano, Alexander (Xander) Farnum, and Lauren Sanchez joined as well.
We are also grateful for our Scientific Advisory Board Member, Dr. Ben Cocanougher, at Cincinnati Children’s, whose passion for working on PURA Syndrome is infectious. It was great to see him present his poster at ASGCT this year and watch him sharing his work on PURA Syndrome with so many.
Thank you for refusing to accept the status quo alongside us. We could not do this without your support!
With deep gratitude,
Kyle & Jean
Jack's Parents
Co-Founders, Jack's Tomorrow
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Consider a donation today and join the Jack’s Tomorrow team as we find and fund a cure for PURA Syndrome.