CNS Delivery in Drug Development and Toxicology: Best Practices and Recent Advances

Central Nervous System (CNS) therapies have significantly progressed in the last decade, propelled by emerging treatment modalities including gene therapies. Neurological indications remain amongst the most challenging to address with longer development timelines and a lower success rate when compared to other therapeutic areas. This session will discuss CNS delivery considerations when using non-clinical models with a focus on best practices. Translational challenges related to biodistribution, species relevance and overall study design will be discussed. The session will also review the status of innovative CNS dosing approaches such as convection enhanced delivery (CED) and focused ultrasound (FUS) in non-clinical models.

Learning Objectives:

1. Understand the range of strategies available when planning central nervous system delivery in preclinical models.

2. Familiarize participants with success factors and sources of variation when conducting central nervous system delivery studies in non-clinical models.

3. Review basic scientific endpoints that are included in preclinical safety studies with central nervous system treatment.