About the symposium
Discovery alone does not change outcomes. Progress happens when scientific insight is translated into coordinated action.
The 2027 St. Jude Pediatric Translational Neuroscience Symposium, taking place April 6–7, 2027, and hosted by the Pediatric Translational Neuroscience Initiative (PTNI), explores how today’s discoveries are becoming tomorrow’s therapies through integrated research, collaborative infrastructure, and innovative translational methodologies.
Over two days, leading investigators from St. Jude and institutions around the world will showcase how discoveries are actively advancing across the translational pipeline—from gene discovery and mechanistic understanding to data integration, therapeutic development, and clinical implementation. The Symposium will spotlight current research, innovative methodologies accelerating progress, and collaborative models that are redefining how therapies are developed for rare pediatric neurological disease.
Centered on the theme Accelerating Translational Action in Pediatric Neuroscience, this event demonstrates how coordinated scientific expertise, multidisciplinary collaboration, and purpose-built translational infrastructure are transforming discoveries into meaningful clinical progress. Researchers, clinicians, data scientists, translational investigators, industry collaborators, and patient advocacy leaders will come together to exchange ideas, share emerging findings, and explore new opportunities to advance therapies for children affected by rare neurological disorders.
This event will be hosted in a hybrid format, giving you the option to attend in person and enjoy enhanced Symposium and networking experiences. As always, we also offer virtual attendance for those whose schedule or location does not allow for in-person attendance.
Whether attending in person or virtually, participants will engage with a dynamic program that reflects the breadth of today’s translational neuroscience landscape while helping shape the next generation of scientific collaboration.
Overview
Event: St. Jude Pediatric Translational Neuroscience Symposium
Date: April 6-7, 2027
Location: St. Jude Children's Research Hospital
Virtual attendance available
Program host:
Pediatric Translational Neuroscience Initiative
Event contact:
PTNI@stjude.org
Session themes
Sessions are organized around the translational research continuum, highlighting the scientific discoveries, collaborative approaches, and innovative methodologies driving progress across pediatric neuroscience.
Topics will include:
- Gene Discovery: Foundations for Translational Action: Exploring emerging approaches to rare disease gene discovery, collaborative genomics, and new scientific insights that establish the foundation for therapeutic development.
- Therapy Development: Precision Medicine through Mechanistic Insights: Examining how mechanistic understanding informs precision therapies through antisense oligonucleotide (ASO) development, advances in AAV technologies, and emerging gene-editing strategies.
- Data Science for Translational Impact: Highlighting the integration of multimodal datasets, computational approaches, and analytical frameworks that strengthen preclinical discovery and accelerate translational decision-making.
- Innovative Pathways Driving Discovery and Translational Progress: Showcasing collaborative scientific models, AI-enabled clinical outcome measures, emerging biological pathways, and next-generation discoveries that are expanding opportunities for therapeutic advancement.
About the St. Jude Pediatric Translational Neuroscience Initiative (PTNI)
The St. Jude Pediatric Translational Neuroscience Initiative (PTNI) is transforming how discoveries become therapies for children living with catastrophic rare neurological disorders. By integrating expertise across laboratory science, clinical research, data science, translational medicine, and strategic partnerships, PTNI has established a coordinated framework that accelerates the path from scientific discovery to patient impact.
Since its official launch in 2020, PTNI has grown into a comprehensive translational neuroscience program that connects every stage of therapeutic development—from gene discovery and disease mechanism to preclinical evaluation, clinical strategy, and experimental therapeutics. Through close collaboration with researchers, clinicians, patient communities, industry, and institutional collaborators worldwide, PTNI is advancing innovative approaches that address some of the most urgent unmet needs in pediatric neuroscience.
As new scientific opportunities emerge, PTNI continues to develop the infrastructure, collaborative models, and translational methodologies needed to move promising discoveries forward with greater speed, rigor, and purpose.
