Post-Conference Delivery Day

Showcasing the Latest Discovery & Research in CNS Administration Routes for Gene Therapies

9:30 am Part 1: An Introduction to CSF & Targeted Delivery

  • Gregory Stewart Executive Vice President, Research & Development, Alcyone Therapeutics

Synopsis

  • Understanding the principles of delivery and distribution
  • Precise and optimal delivery has a profound impact on both efficacy and safety of gene therapy
  • Controlling biodistribution to the targets of interest has the potential to move the frontier of the field by impacting safety, efficacy and potentially the amount of vectors needed for an effective therapy

10:30 am Morning Break

11:00 am Part 2: Intrathecal: Lumbar & Cisterna Magna Administration

  • Steven Gray Associate Professor; Chief Scientific Adviser, University of Texas Southwestern Medical Center; Taysha Gene Therapies

Synopsis

  • Moving away from invasive procedures with intrathecal administration
  • Showcasing translational data to support efficacious biodistribution to brain targets
  • Discussing rare and common disorders compatibility for intrathecal administration

12:00 pm Lunch Break

1:00 pm Part 3: Advancing Intraparenchymal & Intraventricular Delivery Techniques

  • Krystof Bankiewicz Professor of Neurosurgery & Director, Brain Health & Performance Center, The Ohio State University; AskBio

Synopsis

  • Exploring advances in direct administration routes for optimal CNS target engagement
  • Discussing convection enhanced delivery with MRI guidance for maximal cell exposure
  • Overcoming translational challenges with novel devices to ensure consistency and satisfy regulators

2:00 pm CNS Delivery Roundtable Discussion

  • Gregory Stewart Executive Vice President, Research & Development, Alcyone Therapeutics
  • Steven Gray Associate Professor; Chief Scientific Adviser, University of Texas Southwestern Medical Center; Taysha Gene Therapies
  • Heather Gray-Edwards Assistant Professor, Department of Radiology, Horae Gene Therapy Center, University of Massachusetts Medical School

3:00 pm Afternoon Break

3:30 pm Part 4: Next Generation CNS Delivery

  • Heather Gray-Edwards Assistant Professor, Department of Radiology, Horae Gene Therapy Center, University of Massachusetts Medical School

Synopsis

  • Exploring novel administration routes in early research
  • Discussing their suitability to different neurological indications and patient populations
  • Reviewing the recent data in preclinical models for potential translation of new delivery methods for CNS gene transfer including CSF recirculation and subpial injections

4:00 pm Prenatal Gene Therapy for Severe Congenital Disorders

  • Tippi MacKenzie Professor, Surgery; Co-Director, Center for Maternal-Fetal Precision Medicine, University of California, San Francisco

Synopsis

  • Exploring early research for prenatal administration of gene therapies for CNS disorders
  • Advantages of the fetal environment include the ability to induce tolerance to a foreign protein and crossing the blood barrier
  • For some diseases, treating early should improve outcomes

4:30 pm Subpial Cector Delivery for Effective AAV Delivery into Spinal Cord in Adult Animals: Experience in Rodent ALS Models & Large Pre-Clinical Animal Models

  • Martin Marsala Professor, Department of Anesthesiology, UC San Diego School of Medicine

Synopsis

  • Effective wide-spread AAV delivery in the spinal cord of adult animals after spinal subpial delivery
  • Potent disease-modifying effect in pre-symptomatic and symptomatic G93A rats after subpial delivery of shRNA-SOD1
  • Evaluation of human subpial injection device in large animal models

5:10 pm Chair’s Closing Remarks