Novartis disclosed another Phase III failure in muscular dystrophy, as delpacibart etedesiran (del-desiran) missed primary endpoints in the HARBOR trial in myotonic dystrophy type 1. The company said it is evaluating the full dataset to determine next steps, after already facing a separate safety-driven disruption in CAR T development. The renewed readout pressure also highlights how quickly neuromuscular and cell therapy programs can change investor expectations. For the broader sector, it underscores the high bar for efficacy translation in mechanistically complex modalities, including antibody–oligo conjugates.
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