Roundtable Discussion: Enabling Platform-Based Regulatory Strategy for CNS Oligonucleotides
- Reassessing the necessity of fully independent nonclinical toxicology packages for each candidate by examining shared chemistry, mechanism of action, and accumulated class safety data across oligonucleotide platforms
- Identifying the scientific commonalities that could justify a proportionate, risk-based regulatory approach, rather than repeating comprehensive packages where prior pharmacology and toxicology knowledge already exists
- Exploring practical strategies to align regulatory bodies around a platform framework, including early agency engagement, cross-program data integration, and clearer articulation of class-based evidence to enable streamlined, science-driven IND progression