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. 2021 Feb 4;22(4):1561. doi: 10.3390/ijms22041561

Table 1.

Pharmaceutical company-tested therapeutic strategies for Huntington’s disease (HD), using molecules that target huntingtin mRNA that are in clinical testing or close to the start of this phase.

Company Technology/Strategy Molecule Clinical Phase
Ionis Pharmaceuticals/Roche ASO/Non-allele-selective RG6042 I/II—completed
III—scheduled for completion in 2023
Wave Life Sciences/Takeda ASO/Allele-selective WVE-120101, WVE-120102 I/II—scheduled for completion in 2020
UniQure Biopharma RNAi/Non-allele-selective rAAV5-miHTT (AMT-130) I/II—scheduled for completion in 2022
Voyager/Sanofi-Genzyme RNAi/Non-allele-selective AAV-shRNA (VY-HTT01) -
Spark Therapeutics RNAi/Non-allele-selective AAV-shRNA -