UniQure's Gene Therapy Slows Huntington's Disease Progression by 75% in Trials

Researchers have achieved a groundbreaking milestone with the gene therapy AMT-130, developed by uniQure, which has successfully slowed the progression of Huntington's disease by approximately 75% in clinical trials. This one-time treatment involves a neurosurgical infusion of a modified virus that silences the mutant huntingtin (HTT) gene responsible for the disease's toxic protein accumulation, offering a durable clinical benefit unlike previous therapies that required repeated dosing and provided only temporary relief. Trials demonstrated sustained improvements in motor, cognitive, and functional abilities over three years, with treated patients experiencing significantly less decline than those on placebo, potentially extending quality life by decades. The therapy, delivered via a 12- to 20-hour brain surgery and guided by real-time MRI, represents the first treatment to alter the disease's course rather than just alleviate symptoms. uniQure plans to meet with the FDA to discuss accelerated approval, aiming for commercial availability by the end of 2026. This advancement brings new hope to patients and families affected by this fatal genetic disorder and may pave the way for similar gene therapies targeting other neurodegenerative diseases.