A gene therapy for Huntington's disease called AMT-130 may get another chance at FDA approval following the departure of Trump-era officials, including Vinay Prasad, who previously blocked the treatment. The therapy, developed by Dutch company Uni Qure, targets the mutant huntingtin protein that progressively destroys brain nerve cells.

Huntington's disease affects roughly 30,000 Americans, typically striking people in their 40s with no available treatments. Early trial data showed AMT-130 could slow disease progression by up to 75 percent. In 2024, the FDA initially approved filing for accelerated approval without placebo controls, recognizing the ethical dilemma: the treatment requires 10- to 12-hour brain surgery, meaning placebo patients would face sham procedures involving skull drilling.

Prasad's team later reversed this position, demanding sham surgeries despite patient advocacy concerns. A former FDA official called the reversal "truly evil" given the suffering involved. With new leadership in place, the agency appears ready to reconsider the accelerated approval pathway that Uni Qure had originally pursued.

This development could reshape how regulators evaluate gene therapies requiring invasive delivery methods. Patient advocates see renewed hope for a disease that often proves fatal within 15-20 years of symptom onset.