Discovery & Development of PTC518, a Selective Huntingtin Lowering Splicing Modifier for the Treatment of Huntington’s Disease

Time: 4:00 pm
day: Day One


  • Initiated Huntingtin lowering drug discovery program – Identified splicing modifiers and deciphered the mechanism of HTT lowering
  • Lead optimization efforts led to the discovery of PTC518
  • PTC518 data to be shared, demonstrating HTT lowering in cells and animals; POC demonstrated in humans