Advancing Gene-Targeted Therapies for Central Nervous System Disorders

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. This 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 central nervous system (CNS) disorders, and implications of developing these 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. They also discussed what will be needed to develop these therapies for common disorders such as Alzheimer's and Parkinson's disease, as well as neuropsychiatric and neurodevelopmental disorders such as schizophrenia and autism. The workshop included approaches that target both DNA and RNA, as well as gene products using viral vectors, antisense oligonucleotides, and RNA interference. This publication summarizes the presentations and discussion of the workshop. Table of Contents Front Matter 1 Introduction and Overview 2 Exploring the Current Landscape of Central Nervous System Gene-Targeted Therapies 3 Gene-Targeted Therapy Approaches for Central Nervous System Disorders: Opportunities and Challenges 4 Translating Gene-Targeted Therapies from Bench to Bedside 5 Meaningful Engagement of Patients and Families 6 Future Directions in the Development of Gene-Targeted Therapies Appendix A: References Appendix B: Workshop Agenda Appendix C: Registered Attendees