Novel Therapeutics:
Rare Disease Gene Therapy Platform (RDGTx): A Scalable AAV Engine for Pediatric Neurogenetic Disorders
Steven J. Gray, Ph.D.
- Professor, Departments of Pediatrics, Neurology, and Molecular Biology
- Co-Director, UT Southwestern Gene Therapy Program
Berge Minassian, M.D.
- Professor of Pediatrics and Neurology
- Division Chief, Pediatric Neurology
- Co-Director, UT Southwestern Gene Therapy Program
UT Southwestern has built a rare disease gene therapy platform that can take multiple pediatric brain disorders from the lab to the clinic using one shared AAV engine. Instead of developing one drug at a time, this platform reuses the same delivery technology, manufacturing process, and regulatory playbook across many diseases, dramatically lowering cost and risk. The first program, SLC6A1, is fully owned by UTSW and has received strong FDA feedback, placing it on a clear path to first-in-human testing. Once that initial trial is launched, additional rare disease prograM.S. can be “rooftiled” behind it, moving faster and more efficiently than traditional biotech development. This creates a portfolio of high-impact therapies with multiple shots on goal, strong institutional control, and clear commercial optionality.
