Each spring, the American Society of Gene & Cell Therapy (ASGCT) hosts one of the most influential global conferences on gene and cell therapy for rare diseases. With more than 7,500 researchers, clinicians, biotech leaders, regulators, and patient advocates coming together to exchange data, shape ideas, and set the direction of genetic therapies. This is where the future of genetic medicine is taking shape.
FAST was there, making sure Angelman syndrome was part of that future.
What Being in the Room Really Means for Angelman Syndrome
For organizations like FAST, being at ASGCT isn’t a matter of visibility. It’s a matter of relevance. It’s a reflection of where we stand, not just as advocates, but as active contributors to the science, infrastructure, and clinical trial readiness that make Angelman syndrome gene and cell therapies possible.
This year, FAST was one of the only patient advocacy organizations that was invited to deliver multiple talks throughout the conference, a signal that our work is not only recognized, but increasingly relevant to the future of gene and cell therapy. More notably, our Chief Science Officer, Dr. Allyson Berent, was invited to speak in 3 sessions focused on the evolving role of advocacy in scientific advancement. She brought a perspective rooted in experience, the kind that comes from years of building trial infrastructure, supporting biomarker development, and working to translate early-stage research into real-world possibilities, all while understanding the patient and parent’s lived experience. This is a perspective that has been recognized over the past few years at the ASGCT conference.
Her talks underscored something we have long believed: advocacy is not adjacent to science. It's integral to it.
That belief was reinforced throughout the week. In total, there were 10 scientific presentations focused on Angelman syndrome—a mix of posters and talks from FAST-funded researchers, industry collaborators, and our very own chair of our Scientific Advisory Board, Dr. Barbara Bailus. The science was strong. The interest was real. And the presence of Angelman syndrome across multiple sessions was a clear signal that this community is not waiting on the sidelines; we are driving the conversation.
What We Heard and What We’re Watching
One of the most striking themes at ASGCT this year was delivery, specifically, how to cross the blood-brain barrier to deliver gene therapy for neurological disorders like Angelman syndrome. With more than 30 presentations on blood-brain barrier technologies and more than 110 sessions dedicated to rare diseases, it’s clear the field is not only advancing rapidly but actively prioritizing conditions like Angelman syndrome and other ultra-rare neurological disorders. The FAST Science team was able to leverage their incredible scientific expertise to support numerous companies developing these novel capsids, each of whom see Angelman syndrome as the ideal genetic disorder to support their novel technology.
Another was trial readiness. From endpoint development to regulatory engagement, the focus is shifting toward infrastructure: how to design smart, scalable, ethically sound trials in rare and ultra-rare conditions. These are the very areas FAST has been investing in for years: building platforms and partnerships to enable faster progress, and clearer data, meaningful change can be measured, and the learnings are shared and de-siloed amongst all rare neurogenetic disorders.
And throughout the conference, there was a growing awareness that advocacy organizations like FAST are not simply telling stories. We are creating the conditions for science to move.
Laying the Groundwork for What’s Next
We do not attend conferences like ASGCT for the photo op. We show up to ask hard questions, challenge dogmatic assumptions, and bring urgency to conversations that don’t always prioritize rare diseases. For conditions like Angelman syndrome, presence alone isn’t enough, we have to make the science impossible to ignore. We have to be the lowest hanging fruit for technology to be advanced.
Being invited into the conversation means we have earned a level of credibility. Contributing to that conversation means we intend to use it.
We left ASGCT more certain than ever that the science is heading in the right direction—and more committed than ever to making sure that developing meaningful therapeutics for all living with Angelman syndrome stays on that path, not at the margins. FAST’s mission is always at the center of what is possible.