This week, FAST leadership and partners are participating in the American Society of Gene & Cell Therapy (ASGCT) Annual Meeting, one of the leading global forums focused on advancing gene and cell therapy.
This meeting brings together scientists, clinicians, industry leaders, and regulators to share data, debate approaches, and move the field forward. It’s where much of the behind-the-scenes work that shapes future therapies takes place.
Driving Key Conversations at ASGCT
FAST’s presence at ASGCT reflects the role we play in advancing research and supporting the infrastructure needed to move promising therapies forward.
Dr. Allyson Berent Chief Science Officer, FAST Speaker: Patient and nonprofit engagement driving therapeutic advancement Master of Ceremony: ASGCT Momentum Gala
Dr. Berent will speak on how patient advocacy organizations, like FAST, are playing an active role in therapeutic development, from funding early research ideas to helping shape development strategies and bringing promising programs to human application. She will show the power of working alongside our research teams, industry, and regulators to move AS therapeutic research forward.
Her perspective reflects FAST’s approach of staying closely involved at every turn in drug development: from the early science, to supporting the infrastructure needed for progress, and helping align the people and decisions required to move therapies forward responsibly.
She will also serve as Master of Ceremony of the inaugural ASGCT Momentum Gala, the launch of ASGCT’s Foundation, which is bringing together leaders across the gene and cell therapy community.
Ryan Fischer Chief Operating Officer, FAST Workshop Panelist: Collaborating with Clinical Stakeholders - Successes and challenges in clinical engagement
Ryan will participate in a panel examining how patient advocacy organizations work alongside researchers, clinicians, and industry partners to advance early-stage therapeutic development. The discussion will focus on the realities of building these collaborations, including how to assess partnerships, support programs as they move toward first-in-human trials, and navigate the tension between scientific uncertainty and patient expectations.
Dr. Elizabeth Berry-Kravis Director of the RUSH Pediatric Neurosciences F.A.S.T. Center for Translational Research Speaker: Adaptive and innovative study design in rare diseases
Dr. Berry-Kravis will speak on how clinical study design in rare diseases must balance scientific rigor with flexibility, given the realities of small patient populations and complex development pathways.
Advancing the Science Behind What Comes Next
Researchers supported by FAST are also contributing to a wide range of poster presentations at ASGCT.
These studies span multiple conditions, including neurodevelopmental and neurodegenerative disorders as well as other genetic diseases. Much of this work focuses on challenges that apply across gene therapy, including gene editing, delivery to the brain, and the regulation of gene expression.
Researchers include Dr. Beverly Davidson, Dr. Mark Deehan, Dr. Nadav Ahituv, Dr. David Segal, Dr. Yong-Hui Jiang, Dr. Xiaoana Lu, and Dr. Marco Carpenter, whose recent FAST/ASGCT Career Development Award reflects continued investment in emerging leaders in the field.
This broader presence reflects FAST’s commitment to supporting researchers whose work helps move the field forward, even when that work extends beyond Angelman syndrome.
Connecting Angelman Syndrome to the Future of the Field
Advancing therapies for Angelman syndrome depends on more than scientific discovery. It depends on the decisions that determine how that science moves forward.
Which approaches are prioritized.
How studies are designed.
What tradeoffs are made between speed, safety, and feasibility.
In rare diseases, those decisions carry added weight, where small patient populations and limited opportunities make each step critical. This is where FAST plays a role. By staying actively engaged in the field and supporting the work that helps address these challenges, we help ensure that Angelman syndrome is not operating in isolation but is part of the decisions shaping what comes next.