Optimizing Splice-Correcting ASO Dosing & Regulatory Strategies to Target TDP-43–Driven Neurodegenerative Diseases
- Discuss how a comprehensive, data-driven non-clinical development for ASOs can help optimize clinical dosing strategies, for a favorable safety profile, reducing toxicity risks and enabling a more efficient trial design
- Align non-clinical evidence with regional regulatory requirements to facilitate efficient clinical development and access to novel therapeutics for rare disease patients
- Utilize emerging clinical data and real-world insights to refine clinical trial designs and increase chance for early clinical proof-of-concept