Connecting European Neuroscience

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

April 23rd, 2019 to April 24th, 2019
Washington, United States

Genes, Aging, Nervous Systems

About the Event

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.


Board on Health Sciences Policy
National Academies of Sciences 2101 Constitution Avenue, NW Washington, DC 20418


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