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Scientific American Hopes were high for drugs designed to lower levels of a mutant protein, but development has stalled orange  Advertisement Two pharmaceutical companies have halted clinical trials of gene-targeting therapies for Huntington’s disease (HD), following the drugs’ disappointing performance. Researchers had hoped that the treatments known as antisense oligonucleotides (ASOs) would be a game changer for HD, an incurable genetic condition that affects cognition, behaviour and movement. But back-to-back announcements from Roche, headquartered in Basel, Switzerland, and Wave Life Sciences, in Cambridge, Massachusetts, have dealt a crushing blow to those affected by the disease. “I was really shocked, really tearful,” says Marion, a woman in London with HD, who was part of one of the trials. “We didn’t see it coming at all. I felt really frightened and worried about my future.” Marion requested that her last name be withheld to protect her privacy.

United statesSan diegoUnited kingdomCity ofSarah tabriziCath stanleyDon clevelandMike panzaraClaudia testaUniversity college londonUniversity of californiaVirginia commonwealth university in richmondIonis pharmaceuticals in carlsbadHuntington disease associationWave life sciencesDisease association