A gene therapy for Huntington’s disease has been given a new lease of life after the ouster of several Trump officials, particularly Vinay Prasad, who previously rejected the treatment in a move described as “truly evil.”

Huntington’s disease is an inherited condition that typically strikes middle-aged adults. It causes progressive damage to nerve cells in the brain and currently has no cure, leading to early death for many sufferers.

UniQure's AMT-130, a one-time treatment aimed at reducing harmful protein levels, showed promising results in early trials, potentially slowing disease progression by up to 75%. However, the decision on whether to approve the drug hung in the balance due to ethical concerns about conducting sham surgeries for placebo controls.

During Prasad’s tenure as head regulator of gene therapies at the FDA, he demanded that UniQure conduct these invasive sham surgeries. But with his departure and those of other officials, the agency has now reversed its stance, allowing UniQure to move forward without a placebo control group.

This reversal marks not just a victory for patients and their advocates but also a pivotal moment in how regulatory bodies approach complex ethical decisions in medical innovation.