Day One

Tuesday, July 20 2021

Day Two

Wednesday, July 21 2021

*All times are shown in EST

OVERCOMING THE CHALLENGES OF ESTABLISHED & NOVEL ADMINISTRATION ROUTES

8:00 am Explore Digital Platform

8:20 am Next Generation Administration Routes such as Focused Ultrasound

Synopsis

  • Talk details to follow

8:40 am Intracisternal Delivery to the CNS of an AAV Gene Therapy Candidate for the Treatment of GM1 Gangliosidosis

Synopsis

  • Routes of delivery of AAV vectors to the CNS
  • Intracisternal (ICM) administration: bioavailability and safety considerations
  • Preclinical evaluation of ICM-administered AAVrh10 vector carrying human GLB1
  • Status of clinical trial in GM1 gangliosidosis patients

9:00 am Q&A Panel Discussion: Intra-CSF Delivery – Moving from Paediatric to Adult Patient Populations

9:40 am Morning Break & Scientific Poster Session

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 Session Reserved for ClearPoint Neuro

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 Q&A Panel Discussion

2:20 pm Afternoon Break

OPTIMISING NEUROLOGICAL BIOMARKERS & DEVELOPING CLINICALLY RELEVANT OUTCOME MEASURES FOR PATIENTS

2:50 pm Roundtable Discussion: Seeking Translatable Biomarkers for Neurological Disorders

Synopsis

Participate in a focused roundtable discussion to discuss the existing and novel biomarker technologies for a range of neurological disorders.

  • Parkinson’s Disease
  • Alzheimer’s Disease
  • SMA
  • Huntington’s Disease
  • Neurodevelopmental Disorders & Epilepsy

3:20 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

3:40 pm Exploring Genetically Targeted Therapies for Neurodevelopmental Disorders

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

Synopsis

  • Case study illustrating therapeutic strategy, preclinical evidence and biomarker development

4:00 pm Q&A Panel Discussion: Patient Perspectives – Rare to Common Neurological Disorders

  • Grace Liang Vice President, Clinical Development, Neurocrine Biosciences
  • Jay Barth Chief Medical Officer, Lexeo Therapeutics
  • Samir Koirala Director of Research Genetic & Neurodevelopmental Disorders, Biogen

4:30 pm Chair’s Closing Remarks

Day One