HTT-D3: a CNS-Penetrant Molecule with HTT Lowering Activity


HTT splicing modulator PD in Huntington’s disease model from HTS and optimization Nature Communications PTC Therapeutics, So. Plainfield, NJ
Drug Hunter Team

Mutated Huntingtin (HTT) protein is the cause of Huntington’s disease (HD). HTT-D3 is an oral, CNS-penetrant molecule with human HTT lowering activity in vivo used to illustrate the science around PTC Therapeutics’ Huntingtin’s disease program. PTC had filed patent applications for the HD program as early as November 2016 ( WO201700726 ). PTC [...]



Other molecules you may be interested in