PTC Splice Modulators: From a Novel Discovery to a Broad Therapeutic Platform (PTSeek)
- 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