*All times are shown in EST
OVERCOMING THE CHALLENGES OF ESTABLISHED & NOVEL ADMINISTRATION ROUTES
8:20 am Next Generation Administration Routes such as Focused Ultrasound
Synopsis
- Discussing new capsids for IV deliver
- Opening the blood brain barrier with Focused Ultrasound
- Evaluating the different CSF routes for CNS delivery
8:40 am Pre-clinical Intraparenchymal Applications for Gene Therapies in the Non-Human Primate Brain
Synopsis
- Accurate targeting to the deep cerebellar nuclei
- Modifications to accommodate non-human primates
- Benefits of real-time MRI guidance
9:00 am Utilizing Imaging in Support of Gene Therapy Applications
Synopsis
Explore imaging applications in support of gene therapy research
• Discuss advantages and disadvantages of the various imaging modalities
• Present case studies for preclinical multimodality studies to evaluate
pharmacokinetics (PK), biodistribution and gene expression
9:15 am Q&A Panel Discussion: Intra-CSF Delivery – Moving from Paediatric to Adult Patient Populations
9:45 am Morning Break
TRANSLATING NEUROSURGICAL DELIVERY TO CATER FOR DIFFERENT PATIENT POPULATIONS
10:40 am Neurosurgery Considerations: Moving from Rare to Common Patient Populations
Synopsis
- Exploring the technical aspects in stereotactic surgery for neurodegenerative disorders
- Evaluating different stereotactic techniques, pros and cons for convection enhanced delivery
- Highlighting pitfalls and complications, avoidance strategies and accuracy (technical limitations vs. advertisement)
11:00 am Target Specific, Tailored Catheter Geometry: Facilitating Optimum Catheter Placement & Associated Infusion Performance for Delivery of Gene Therapies
Synopsis
How catheter design can positively affect versatility for both catheter
placement and infusion distribution characteristics.
• Utilising the presence of the infusion reflux phenomena to influence functional
performance of the intraparenchymal infusion mechanism. A design attribute
that enables the manipulation of a desired distribution pattern to best-fit the
chosen target structure.
• Using a common platform for multiple indications: One-off, acute infusions
through standard connectors or long-term chronic, intermittent reaccess
infusions via a novel transcutaneous port
11:30 am Q&A Panel Discussion: Overcoming Neurosurgical Challenges for Gene Therapy Delivery
12:30 pm Lunch & Networking
OVERCOMING CLINICAL CHALLENGES TO PROVIDE VALUE FOR PATIENTS
1:30 pm Overcoming CNS Immunogenicity with Gene Therapy
Synopsis
- Highlighting immunogenic challenges specifically for CNS disorders
- Measuring the immunogenic responses in the brain
- Discussing mitigation strategies
1:50 pm AAV Gene therapy targeting APOE4-associated Alzheimer’s Disease
Synopsis
- Discussing the basis for a gene therapy approach in an Alzheimer’s Disease population
- Considerations for Phase 1 study design and use of biomarkers
- Status of clinical trial in APOE4 homozygotes with Alzheimer’s Disease
2:10 pm Exploring Genetically Targeted Therapies for Neurodevelopmental Disorders
Synopsis
- Angelman Syndrome as a case study – therapeutic strategies, preclinical evidence and biomarker development
2:30 pm Q&A Panel Discussion
3:00 pm Chair’s Closing Remarks
