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

Time: 9:00 am
day: Conference Day One

Details:

  • 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

Speakers: