PTC518 Mediated Splice-Induced HTT Lowering: Pathway from Concept to Patients
Time: 9:30 am
day: Day Two
Details:
- Uncovering how animal models demonstrated that reducing mutant huntingtin protein (mHTT) levels could alleviate Huntington’s disease (HD)-like symptoms
- Reviewing the discovery and development of PTC518, a small molecule splicing modifier that reduces the production of the mHTT protein in cells and animals
- Highlighting PTC518’s clinical data to demonstrate proof of mechanism and dose-dependent lowering of HTT mRNA and protein levels in healthy volunteers and HD patients
