Huntington's hope diminished after early-stage safety stumble

The outlook for a much-anticipated innovative treatment for Huntington’s disease has been muddied by a surprise serious side effect in a Phase I/II trial.

Following the adverse event, Dutch gene therapy company uniQure (Nasdaq: QURE) said it was stopping work on the higher-dose cohort in the trial, which is studying the gene therapy AMT-130.

Shares in the company slumped over a quarter on Monday, and continued to fall after the closing bell, despite the firm’s reassurances that the lower-dose cohort was unaffected and that “no impact is expected on anticipated data readouts in 2023.”