Pre-Conference Workshop Day

Workshop A: Measuring & Mitigating CNS Immunogenicity

Monday, 19 July - 7.00AM - 5.00PM EST

With the delivery of gene therapy to the CNS, there are associated immunogenic responses, whether induced by the transgene or vector. This workshop will provide a comprehensive introduction into the different types of immunogenicity found in the CNS, whilst looking at the most novel data in the space.

Attend this workshop to:

  • Gain a better understanding of the different types of immunogenicity detected in the CNS with gene therapy administration
  • Discuss the methods for the measurement and evaluation of immunogenicity levels in the brain
  • Explore the different strategies for mitigating toxicity and immunogenic repsonses in the brain
  • Debate immunosuppression and re-dosing challenges for different patient populations
Manuela Corti - Florida

Manuela Corti
Assisant Professor
University of Florida

Workshop B: Understanding the Translational & Biocompatibility Challenges of Intraparenchymal Delivery

With options for either traditional or MRI guided techniques for intraparenchymal delivery of gene therapies to the brain, there are plenty of challenges with in vivo study design, clinical holds and translating from in vivo studies to different clinical settings. Discussing the limitations of current cannula systems both clinically and in non-human primates (NHP), this workshop will focus ondesigning biocompatibility studies and overcoming the time intensive and costly impacts of current technology capabilities.

Attend this workshop to:

  • Discover translational and reverse translational strategies for CNS targeted delivery
  • Consider NHP animal procurement, biodistribution and dosage extrapolation studies
  • Optimise the process and workflow of both traditional vs MRI guided - addressing cost and time limitations
Stephane Palfi - Paris

Stephane Palfi
Professor & Head of
Henri Mondon Medical
Center, Paris University

Workshop C: Gene Therapy for Neurodevelopmental Disorders

Targeting neurodevelopmental disorders with gene therapies comes with very specific challenges attributed to the heterogeneity of the disorders and patients themselves. This workshop will introduce the exciting preclinical research focusing on targeting neurodevelopmental disorders such as Autism and other related conditions, highlighting the opportunity whilst discussing the field’s hurdles.

Attend this workshop to:

  • Hear about the early research being done in gene therapy for neurodevelopmental disorders
  • Explore the preclinical, translational and clinical challenges associated with these patient populations
  • Debate gene therapy viability as a therapeutic approach for these disorders

Suyash Prasad
Chief Medical Officer
& Head of Research &
Taysha Gene Therapies