Advancing Gene-Targeted Therapies for Central Nervous System Disorders: A Workshop

When: April 23, 2019 - April 24, 2019 (1:30 PM Eastern)
Where: National Academy of Sciences Building (The Lecture Room) • 2101 Constitution Avenue, NW, Washington, DC 20418

Topics Biomedical and Health Research, Diseases, Aging, Substance Use and Mental Health, Quality and Patient Safety, Public Health
Activity: Forum on Neuroscience and Nervous System Disorders
Board: Board on Health Sciences Policy


Register for the Workshop

On April 23 and 24, 2019, the Forum on Neuroscience and Nervous System Disorders will host a public workshop that will bring together experts and key stakeholders from academia, government, industry, and non-profit organizations to explore approaches for advancing the development of gene-targeted therapies for central nervous system (CNS) disorders, including approaches that target nucleic acids, such as adeno-associated viruses (AAVs), antisense oligonucleotides (ASOs), and RNA interference, as well as gene product-targeted therapies.

Invited presentations and discussions will be designed to:

  • Provide an overview of the current landscape of gene-targeted therapies approaches for nervous system disorders.
  • Discuss lessons learned from recent advances in gene therapy and ASO development for retinal dystrophy and spinal muscular atrophy.
  • Compare features of different gene-targeted therapy approaches in development for CNS disorders, and discuss approaches to matching the approach to specific diseases, addressing their respective administration, distribution, and dose challenges, and potential long-term effects.
  • Explore clinical development—including biomarker and clinical endpoint selection, trial design to demonstrate disease modification, and the regulatory path—for gene-targeted therapy approaches for rare genetic disorders that have more variable onset and slower progression.
  • Discuss what it would take to move beyond rare genetic disorders to develop gene-targeted therapy approaches for more common, heterogeneous disorders such as Alzheimer’s and Parkinson’s diseases.
  • Explore opportunities for catalyzing development of gene-targeted therapy approaches for CNS disorders, including potential collaborative efforts among sectors and across disorders.

Previous Meetings for this Activity

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