CONFERENCE DAY TWO - 22nd NOVEMBER 2024
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
Synopsis
- 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
Synopsis
- 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
Synopsis
- 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
Synopsis
- 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
Synopsis
- 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
Synopsis
- 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
Synopsis
- 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
Synopsis
- 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