Dr. Allyson Berent on Angelman Syndrome, FAST, GTX-102 & Parent-Led Drug Development

In this GENEration Hope interview, Ron Kleiman speaks with Dr. Allyson Berent — rare disease mom, veterinarian, interventional specialist, researcher, biotech founder, and Chief Science Officer of FAST, the Foundation for Angelman Syndrome Therapeutics. After her daughter Quincy was diagnosed with Angelman syndrome, Allyson became one of the leading voices in parent-led drug development. Through FAST, she helped drive research programs that have moved from family urgency to real clinical development, including GTX-102, which is now in a fully enrolled global Phase 3 study, with data expected in the second half of 2026. In this conversation, we talk about Angelman syndrome, FAST, GTX-102, AS2 Bio, gene-targeted therapies, the realities of building treatments for rare diseases, and what happens when families refuse to wait for someone else to move the science forward. We also discuss the broader future of rare disease treatment — including ASOs, gene replacement, gene editing, stem-cell-based approaches, clinical trials, access, urgency, and the role families play in changing what is possible. If conversations like this are important to you, please subscribe, like, and share. It is the best way to support GENEration Hope and help us reach more families, researchers, clinicians, and advocates. Visit GENErationHope.co to watch more interviews, sign up for the newsletter, and follow our weekly updates on gene therapy, RNA therapy, newborn screening, clinical trials, and rare disease research. Subscribe to GENEration Hope on YouTube for more interviews and weekly updates. Disclaimer: This interview is for educational and informational purposes only and should not be taken as medical advice. Families should speak with their own physicians, specialists, or clinical trial teams about any treatment or research questions. #AngelmanSyndrome #RareDisease #GeneTherapy #RNATherapy #GTX102 #FAST #GenerationHope