Pre-Conference Workshop Day

Workshop C: Gene Therapy for Neurodevelopmental Disorders

8.00am - 10.00am EST

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

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

11.00am - 1.00pm EST

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