How close are we to real breakthroughs in Fragile X research? That’s the question driving FRAXA’s work, and the focus of our latest webinar, now available to watch in full.
On June 26, 2025, FRAXA Co-Founders Dr. Mike Tranfaglia and Katie Clapp hosted a live webinar unveiling the latest research grants funded this year. This fast-paced, info-packed session covered 16 new projects from researchers around the world, each working to better understand, treat, or even cure Fragile X syndrome.
From cutting-edge ASO (antisense oligonucleotide) therapy and gene editing to personalized organoids and real-world behavioral tracking in mice, these projects reflect where the science is heading and why there’s more reason than ever to be hopeful.
2025 Grants Driving Fragile X Research Forward
ASO Therapy Moves Forward
One of the biggest developments comes from Dr. Joel Richter’s lab at UMass Chan, whose groundbreaking ASO work is now being commercialized by biotech company CureAlis. FRAXA continues to fund Richter’s lab to refine biomarkers that will support future human trials.
New Tools to Test Treatments
Quiver Biosciences is developing a medium-throughput testing platform using optogenetics, allowing researchers to see in real time how different Fragile X cell lines respond to therapies.
Gene Reactivation and Editing
Dr. Jeannie Lee’s lab at Harvard and Dr. He Li in Texas are exploring innovative ways to reactivate or replace the silenced Fragile X gene, with promising early results in organoids and mice.
Organoids Take Center Stage
Several labs including those at Emory, the University of Cincinnati, and internationally are using patient-derived brain organoids to test therapies in a more personalized, human-relevant way.
Behavioral Breakthroughs
A Belgian team is using RFID-equipped live mouse tracking to monitor complex social behaviors, offering a powerful new way to evaluate treatments in animal models.
Understudied but Important Areas
This year’s grants also include research into mitochondrial dysfunction, white matter changes, RNA methylation, and sex differences in Fragile X. These are areas with significant potential that have historically received less attention.
Real Questions, Real Emotions: A Candid Community Q&A
The session wrapped with a live Q&A where families and researchers asked thoughtful, heartfelt questions. One moment that stood out: a parent asked whether any of this will make a difference in her son’s lifetime.
Mike and Katie responded with honesty. Some treatments are closer than ever, especially if ongoing clinical trials show success this year. Others may take longer, but progress is happening, and your support is accelerating it.
Together, we’re building the path to better treatments and, one day, a cure.
