Advancing Gene-Targeted Therapies for Central Nervous System Disorders: Proceedings of a Workshop


Note: Proceedings contain the opinion of the presenters, but do NOT reflect the conclusions of the Health and Medicine Division or the National Academies. Learn more about the differences between Reports and Proceedings.

On April 23 and 24, 2019, the Forum on Neuroscience and Nervous System Disorders convened a workshop titled Advancing Gene-Targeted Therapies for Central Nervous System Disorders in Washington, DC. The public workshop brought together experts and key stakeholders from academia, government, industry, philanthropic foundations, and disease/patient-focused nonprofit organizations to explore approaches for advancing the development of gene-targeted therapies for CNS disorders, including approaches that target nucleic acids, such as adeno-associated viruses, antisense oligonucleotides, and RNA interference, as well as gene product-targeted therapies. Participants explored lessons learned from both successful and unsuccessful clinical development programs; new knowledge about the genetic underpinnings of brain disorders; the current status and future potential of gene-targeted therapies for CNS disorders; challenges and potential solutions for translating preclinical findings to approved therapies; and patient and caregiver perspectives.