Home | Central Nervous System | Huntington’s disease | Congress activities | SpliceCon | SpliceCon 2021 poster: Small molecule splicing modifiers with systemic HTT-lowering activity

SpliceCon 2021 poster: Small molecule splicing modifiers with systemic HTT-lowering activity

Data from preclinical studies of investigational small molecule splicing modifiers as potential treatments for Huntington’s disease are shown in our poster presented at the virtual SpliceCon 2021 meeting 

  • Results are presented from analysis of orally bioavailable small molecule splicing inhibitors in vitro and in murine models of Huntington’s disease
  • A novel splicing mechanism for HTT pre-mRNA in vivo is presented, alongside regulatory and selectivity mechanisms for the molecules under investigation
  • In vivo measurements of HTT levels in mouse models are detailed and a proposed Phase 1 clinical trial is discussed
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MED-ALL-HD-2100025 | October 2021
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