8:00 am Check In & Coffee

8:50 am Chair’s Opening Remarks

Moving a CNS Gene Therapy into the Clinic

9:00 am Determining Appropriate Clinical Endpoints for CNS Gene Therapies to Treat Neurodegenerative Disorders


  • Successfully detecting significant changes in a slow-progressing disease within the timeframe of a clinical trial
  • Utilizing a dual endpoint strategy to combine surrogate and clinical endpoints to gain a comprehensive picture of the gene therapies impact in the CNS
  • Discussing novel techniques to analyze patients and define specific patient subgroups, disease status

9:30 am Roundtable: Clinical Lessons Learnt from RNA and ASO Approaches in the CNS


  • Successfully proving clinically viable biodistribution and potential biomarkers
  • Considerations when maintaining efficacy and safety for advanced CNS directed therapies
  • Outlining experiences with regulatory bodies during clinical trials

10:00 am Morning Refreshments

Discussing the Ideal Route of Administration for CNS Gene Therapies

10:30 am MRI-Guided Administration of Gene Therapies to the Brain


  • Discussing the importance of MRI administration in direct brain delivery
  • Assessing the long-term safety of MRI-guided administration
  • Future directions for MRI guided delivery to the brain

11:00 am Panel Discussion & Presentation: Focused Ultrasound Mediated AAV Gene Therapy Delivery to the CNS

  • Isabelle Aubert Senior Scientist, Sunnybrook Research Institute
  • Frederic Padilla Director of the Gene and Cell Therapy Program, Focused Ultrasound Foundation


  • Using ultrasound mediated delivery to open the BBB and drive the successful delivery of capsids into the CNS
  • Outlining the advantages and disadvantages of using MRI guided trials to translate this technology successfully into humans
  • Discussing the therapeutic potential for novel vectors to be used in conjunction with focussed ultrasound technology for optimal delivery

12:00 pm Intraparenchymal Delivery of Gene Therapy for Parkinson’s Disease


  • Discussing the benefits of using targeted delivery methods for CNS gene therapies
  • Ensuring durable gene expression in the brain using intraparenchymal delivery
  • Discussing the biodistribution achieved using intraparenchymal delivery

12:30 pm Lunch & Networking

Assessing the Potential Toxicity of Gene Therapy in the CNS

1:30 pm Successfully Scaling CNS Gene Therapy Dosage when Translating into NHPs

  • Archana Belle Vice President - Translational Research, Encoded Therapeutics


  • Outlining the limitations of allometric scaling for CNS directed gene therapies when translating from rodents to NHPs
  • Discussing strategies to maintain efficacy of transduction during translational studies
  • Employing a layered approach and harnessing PBPK modelling to create a comprehensive model for dose prediction in NHPs to maintain safety and efficacy

2:00 pm Early Toxicology Studies for AAV Gene Therapy to Treat Friedreich’s Ataxia


  • Findings from preclinical toxicology studies evaluating the safety profile of AAV-based gene therapy for Friedreich’s Ataxia
  • Leveraging preclinical findings to guide dose selection, identify potential safety concerns, and optimize treatment strategies for human patients
  • Explore critical data on potential vector-related toxicities, biodistribution patterns, and long-term effects in relevant animal models

2:30 pm Afternoon Refreshments

Future Considerations for CNS Gene Therapies for Larger Patient Populations

3:30 pm Panel Discussion: Making Gene Therapies for Common CNS Diseases a Reality


  • Designing gene therapy vectors to effectively target brain regions and cell types on more common diseases like AD, PF and Huntington’s
  • Leveraging non-viral vectors to improve accessibility and efficacy of gene therapies in the CNS
  • Discussing the role that gene therapy will play in the future of CNS disease management and potential cures
  • Using Genetic Populations to Validate Clinical Success Before Applying to Larger Patient Populations

4:15 pm Chair’s Closing Remarks

4:20 pm End of Conference