Gene Therapy AMT-130 Shows Promising Results in Slowing Huntington's Disease Progression

A groundbreaking gene therapy, AMT-130, has demonstrated remarkable potential by significantly slowing the progression of Huntington's disease by 75% in a pivotal Phase I/II clinical study, offering substantial hope for patients with this devastating neurodegenerative disorder. However, despite these promising results, the U.S. FDA recently indicated that the external control data from these studies are no longer considered adequate for a Biologics License Application, introducing uncertainty into its approval timeline.