Roche ends two Huntington's gene-silencing programs after disappointing data
Roche is ending two gene-silencing programs aimed at Huntington’s disease after disappointing clinical data. Gene silencing seeks to lower production of the mutant huntingtin protein that drives the inherited, fatal neurodegenerative disorder — an approach that has drawn years of investment but has yet to yield an approved therapy.
Roche has been one of the most prominent players pursuing that strategy in Huntington’s, and shutting down two programs removes a significant share of the late-stage pipeline for a disease with no treatments that slow its progression. The decision underscores how difficult it has been to convert a biologically compelling target into a drug that measurably helps patients, and it narrows the near-term options for families waiting on the approach.