Post-Conference Delivery Day

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

9:30 am Gene Therapy Vs the Brain: It Won’t Work, if it Doesn’t Get There

  • Gregory Stewart Executive Vice President, Research & Development, Alchemy Neuroscience

Synopsis

  • Delivery and distribution, the Achilles heal of translation
  • If you could cross the blood brain barrier, would you really want to?
  • Benefits of direct vector delivery to the CNS
  • Optimizing gene therapy through targeted drug delivery to the CNS

10:00 am Case Study: Long-term, Direct, Intermittent CED for Drug Development Studies

  • Owen Lewis Technical Manager, Drug Delivery Device Research & Product Development, Renishaw

Synopsis

  • Exploring the use of a translational drug delivery platform to deliver single or
    multiple doses immediately after surgery or at distant time points
  • Share our experience in the implementation of a 12month repeat dose study in the
    rhesus model
  • Discuss applications for gene therapy as well as other metabolized or short halflife
    molecules.

10:30 am VIRTUAL: Intrathecal: Lumbar & Cisterna Magna Administration

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

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

11:15 am 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

12:00 pm VIRTUAL: Prenatal Gene Therapy for Severe Congenital Disorders

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

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

12:30 pm Lunch Break & CNS Delivery Roundtable Discussion

  • Gregory Stewart Executive Vice President, Research & Development, Alchemy Neuroscience
  • Heather Gray-Edwards Assistant Professor, Department of Radiology, Horae Gene Therapy Center, University of Massachusetts Medical School
  • Chris Tarapata Vice President, Platform Lead, Device Development, Akouos
  • Massimo Fiandaca Executive Vice President, Therapeutic Strategies, CNS Gene Therapy, AskBio

2:00 pm Advancing Intraparenchymal & Intraventricular Delivery Techniques

  • Massimo Fiandaca Executive Vice President, Therapeutic Strategies, CNS Gene Therapy, 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

2:30 pm VIRTUAL: 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
    (V)

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

3:15 pm Chair’s Closing Remarks

  • Gregory Stewart Executive Vice President, Research & Development, Alchemy Neuroscience

(V) = Speaker Presenting Virtually