*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

  • Megan Keiser Project Manager, Translational Neuroscience & Director, Laboratory Research, The Children’s Hospital of Philadelphia

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

  • Jan Vesper Professor of Neurosurgery, Heinrich-Heine- University

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

  • Max Woolley Technical Fellow, Drug Delivery, Renishaw

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

  • Krystof Bankiewicz Professor, Neurosurgery, The Ohio State University
  • Stephane Palfi Professor & Head of Neurosurgery, Henri Mondor Medical Center, Paris University (UPEC)
  • Jan Vesper Professor of Neurosurgery, Heinrich-Heine- University
  • William Gray Professor of Neurosurgery, Cardiff University

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

  • Jay Barth Chief Medical Officer, Lexeo Therapeutics

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

  • Samir Koirala Director of Research, Genetic & Neurodevelopmental Disorders Unit, Biogen

Synopsis

  • Angelman Syndrome as a case study – therapeutic strategies, preclinical evidence and biomarker development

2:30 pm Q&A Panel Discussion

  • Jay Barth Chief Medical Officer, Lexeo Therapeutics
  • Samir Koirala Director of Research, Genetic & Neurodevelopmental Disorders Unit, Biogen
  • Federico Mingozzi Chief Scientific Officer, Spark Therapeutics

3:00 pm Chair’s Closing Remarks