Gene therapy for Huntington’s disease has a new path to approval by the Food and Drug Administration after the dismissal of several Trump administrations, notably. Vinay Prasad

Gene therapy company UniQure has developed a one-time treatment, AMT-130, which aims to lower in the brain the mutant protein that causes the disease, called huntingtin. Data from a small, early trial suggested the drug could

In 2024, the FDA indicated to UniQure that it could file for accelerated approval of AMT-130 without a placebo control arm in its trial. While having a placebo control offers a high-quality comparator in a trial, it raises unique ethical concerns for UniQure’s gene therapy. AMT-30 delivery requires 10 to 12 hours of brain surgery, which means the placebo arm of the trial would require patients in the control group to undergo a lengthy surgery that would involve drilling a visible hole in their skulls.

UniQure went ahead without a placebo control, using outpatient, untreated patients as a comparator control group for their trials, believing the FDA approved the plan. But during Prasad’s tenure as the FDA’s head regulator of gene therapies, the agency tossed the agreement and asked UniQure to perform fake surgeries such as controls.