The 2025 SLC6A1 Connect Symposium was held on December 4th, just before the annual American Epilepsy Society Conference, in Atlanta, Georgia. The symposium was attended by SLC6A1 researchers and families.
Opening and Gene Therapy Announcement, Amber Freed, Founder, SLC6A1 Connect
Patient Perspective - Riley's Experience
Speaker: Will Cheesebro
Institution: SLC6A1 Connect
A Targeted Approach for Gene Therapy
Speaker: Henry Lee, PhD
Institution: Galibra Neuroscience
Cell Type-Specific AAV Gene Therapy
Speaker: Rong Guo, PhD
Institution: Allen Institute
Towards the Development of Safe and Effective Gene Therapy
Speaker: Simone Borsani, PhD Student
Institution: Children's Medical Research Institute, Australia
Improved AAV9 Variant for Enhanced Brain Transfer
Speaker: Steve Gray, PhD
Institution: UT Southwestern
One and Done: Agile CMC for N of 1
Speaker: Susan D'Costa, PhD
Institution: Genezen
SLC6A1 Discovery Snapshot
Speakers: Gay Grossman, Amanda Singleton CGC
Institution: GeneDx
Panel: Considerations of Gene Therapy
Moderator: Emma James, PhD
Institution: Encoded Therapeutics
Developing a Quantitative EEG-Based Biomarker
Speakers: Jacob Tiller
Institution: Brown University and SLC6A1 Connect
Clinical Signature and Longitudinal Seizure Pattern
Speakers: Dr. Pamela McDonnell, Laina Lusk Stripe MMsc
Institution: Children's Hospital of Philadelphia
State and Future Direction of the Natural History Study
Speakers: Dr. Marie Varnet
Institution: Children's Hospital Colorado
Ravicti Clinical Trial Update
Speakers: Dr. Zachary Grinspan
Institution: Weill Cornell Medical School
More than Just Seizures Discussion: Addressing Family Concerns
Speakers: Dr. Scott Demerest
Institution: Children's Hospital Colorado
Panel: Discovery to Gene Therapy > 10 Years, How Do We Catch Up
Moderator: Dr. Anup Patel
Institution: Nationwide Children's Hospital
The Ravicti Conundrum - Balancing Benefit vs Access Headache
Speakers: Amber Freed
Institution: SLC6A1 Connect
Comparing the Forms of Butyrate
Speakers: Katty Kang, PhD
Institution: Vanderbilt University
Panel: Small Molecule Discussion
Moderator: Dr. Michael Rogawski
Institution: University of California, Davis
Exploring Molecular Mechanisms of GABA Dysregulation in Drosophila Models
Speakers: Paige Hall, Julie Cohen
Institution: Baylor College of Medicine. Tulane School of Medicine
A Human iPSC-Derived Medial Ganglionic Eminence Organoid Model
Speakers: Maria Carmen Varela, PhD Candidate
Institution: University of Michigan
Pathologic GABAergic Neurotransmission in Slc6a1(S295L) Mice
Speakers: Kirill Zavalin, PhD
Institution: Vanderbilt University
Panel: Encouraging Industry Partnership & Investment
Moderator: Terry Jo Bichell, PhD
Institution: COMBINEDBrain
Closing Remarks: Parent Perspective
Speaker: Stuart Place
Institution: SLCA1Connect, Australia