Matt Woll

• Sequence specific splice modulators have demonstrated therapeutic application for SMA and HD
• PTC has designed a discovery platform, PTSeek, that couples proprietary splicing data analysis tools with a unique library of small molecules
• Several novel series of molecules are being advanced to influence the expression of disease relevant genes through splicing modulation
• Novel splicing modulators target 5’ splice site sequences that are differentiated from the standard “GA-space” occupied by SMA and HD compounds

• How can expanding the target space beyond the common splice modulators unlock new opportunities for targeting novel disease areas

• What strategies are enabling the design of chemically diverse scaffolds to overcome current limitations in the ligand design space

• Are we overdependent on nonsense-mediated decay and can reducing this reliance drive broaden therapeutic applicability of splice modulators

• What lessons can be drawn from recent investments and platform development to guide future innovation in RNA-targeted small molecules