A prominent gene therapy company with offices in Europe and the US has announced a groundbreaking therapy for Huntington’s disease that slowed disease progression by 75% in human clinical trials.
Huntington’s disease is an inherited neurodegenerative disease, caused by a DNA mutation in the huntingtin gene, that leads to abnormal accumulation of the huntingtin protein in the brain, as well as motor impairment and cognitive decline.
AMT-130, the gene therapy in two trials led by the uniQure company, consists of delivering a virus that contains instructions for the brain cells to block huntingtin protein production in Huntington’s patients. Because brain cells are permanent, the therapy should be unending.
In 29 patients, AMT-130 was well-tolerated for three years after the surgery and prevented the accumulation of the abnormal protein in the brain, decreasing degeneration and delaying disease symptoms.
“My patients in the trial are stable over time…, and one of them is my only medically retired Huntington’s disease patient who has been able to go back to work,” said Ed Wild, clinical researcher at University College London.
This breakthrough was only possible due to numerous years of preclinical research, including studies in brain cells from Huntington’s patients and studies using mice, pigs, rats and monkeys, essential to inform the design of the therapy and its efficacy.
The company aims to launch the drug in the US by the end of 2026 and then in Europe.