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molecules of the month


HTT splicing modulator

PD in Huntington’s disease model

from HTS and optimization

Nature Communications

PTC Therapeutics, So. Plainfield, NJ

Chemical structure of molecule HTT-D3
2 mins read

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 has an oral, CNS-penetrant molecule for Huntingtin disease program in Phase II (PTC518). Initial data from the Phase I study showed HTT mRNA and protein lowering in human healthy volunteers as well as PTC518 exposure in the CNS. There are no approved disease-modifying treatments for HD, and emerging strategies targeting HTT such as RNAi and ASOs currently cannot penetrate the CNS without an invasive procedure. Treating Huntingtin’s would likely…

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