Welcome to the 3rd Annual Gene Therapy for Neurological Disorders

Over 4 comprehensive days of networking, presentations and discussion sessions, the 3rd Gene Therapy for Neurological Disorders meeting will enable you to:

  • Discuss the latest DRG toxicity and immunogenicity research to understand the biology behind AAV toxicity in the CNS
  • Evaluate current safety and toxicity strategies to navigate off target effects including redosing options
  • Evaluate intraparenchymal, intra-cisterna magna, lumbar intrathecal and systemic delivery routes for CNS indications to assess CNS tropism and biodistribution
  • Hear from neurosurgeons at the forefront of delivery and explore the most up to date delivery devices to identify novel innovations and areas for development
  • Review pharmacological concepts for preclinical dose selection in NHP biodistribution case studies to optimize dose extrapolation
  • Learn about the different vector discovery strategies used to identify vectors with improved CNS tropism and specificity
  • Optimize clinical trial design considerations for both paediatric and adult neurological patient populations to showcase efficacy to regulators and deliver value for patients
  • Discover the novel indications being targeted by gene therapies such as epilepsy, neuropathic pain and hearing disorders to improve your knowledge of current CNS pipelines