Table 1.
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 | - |