Reversed “Truly evil” FDA rejection of gene therapy overturned after Trump official ousted Gene therapy company UniQure had another FDA meeting after Vinay Prasad’s exit. Beth Mole – Jun 17, 2026 11:12 am | 113 The Food and Drug Administration headquarters in White Oak, Maryland. Credit: Getty | Congressional Quarterly The Food and Drug Administration headquarters in White Oak, Maryland. Credit: Getty | Congressional Quarterly Text settings Story text Size Small Standard Large Width * Standard Wide Links Standard Orange * Subscribers only Learn more Minimize to nav A gene therapy for Huntington’s disease has a new path toward approval from the Food and Drug Administration after the ouster of several Trump officials, particularly Vinay Prasad , who rejected the therapy in a shocking move one former FDA official called “truly evil.” Huntington’s disease is an inherited condition that typically strikes in middle age and causes nerve cells in the brain to gradually break down. There are currently no treatments for the disease, and many afflicted die in their 50s and 60s. Gene therapy company UniQure developed a one-time treatment, AMT-130, that aims to lower brain levels of the mutant protein behind the disease, called huntingtin.
Data from a small, early trial suggested the drug could slow the progression of the disease up to 75 percent , and patients and advocates have closely watched the drug’s development in hopeful anticipation. 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. Delivery of AMT-30 requires a 10- to 12-hour brain surgery, which means a placebo-control arm of a trial would require patients in a control group to undergo a lengthy sham surgery that could involve drilling a superficial hole in their skulls.
Source: https://arstechnica.com/health/2026/06/truly-evil-fda-rejection-of-gene-therapy-overturned-after-trump-official-ousted/
Data from a small, early trial suggested the drug could slow the progression of the disease up to 75 percent , and patients and advocates have closely watched the drug’s development in hopeful anticipation. 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. Delivery of AMT-30 requires a 10- to 12-hour brain surgery, which means a placebo-control arm of a trial would require patients in a control group to undergo a lengthy sham surgery that could involve drilling a superficial hole in their skulls.
Source: https://arstechnica.com/health/2026/06/truly-evil-fda-rejection-of-gene-therapy-overturned-after-trump-official-ousted/