An ad hoc committee will plan and conduct a 1.5-day 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, antisense oligonucleotides, 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 therapy approaches for CNS 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 progression.
• Discuss what it would take to move beyond rare genetic disorders to develop gene-targeted therapy approaches for 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.

The planning committee will develop the agenda for the workshop, select and invite speakers and discussants, and moderate the discussions. A proceedings of the presentations and discussions at the workshop will be prepared by a designated rapporteur in accordance with institutional guidelines.