Mechanism of Action
An antisense oligonucleotide (ASO) delivered by intrathecal injection that binds HTT mRNA and triggers its degradation, reducing production of both mutant and normal huntingtin protein non-selectively. After an earlier Phase 3 trial (GENERATION HD1) was halted for lack of benefit at the studied dose/frequency, Roche is now evaluating a lower, less-frequent dosing regimen in younger patients with less disease burden in a new Phase 2 trial (GENERATION HD2).