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Double setback for ASO trials in Huntington disease

Clinical failures of antisense candidates from two companies highlight the challenges for huntingtin-lowering approaches, but a diverse pipeline could yet provide a disease-modifying therapy.
  1. Katie Kingwell
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The failures of Roche and Wave Therapeutics’s antisense oligonucleotide (ASO) candidates in Huntington disease (HD) have left the community in mourning. Whereas Roche’s phase III drug tominersen worsened outcomes and may have overshot the activity mark in the neurodegenerative disease, Wave’s phase I/II candidates appeared to miss their targets, investigators reported at an HD meeting in late April.

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Nature Reviews Drug Discovery 20, 412-413 (2021)

doi: https://doi.org/10.1038/d41573-021-00088-6

Updates & Corrections

  • Clarification 20 May 2021: The article has been updated to clarify the status of the trials of antisense oligonucleotides developed by Wave Therapeutics and the name of CHDI.

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