UK Researchers Develop Gene Therapy to Slow Huntington's Disease by 75%

What's Happening?

Researchers in the United Kingdom have announced a significant breakthrough in the treatment of Huntington's disease, a fatal neurodegenerative disorder. The new gene therapy, AMT-130, has shown the potential to slow disease progression by up to 75% over three years. This therapy involves injecting a high dose of AMT-130 into the brain, where neurons absorb the custom-made DNA, which prevents the production of the mutant huntingtin protein responsible for the disease. The trial, which involved 29 patients, demonstrated that those receiving the high dose experienced significantly less disease progression compared to those who did not receive the treatment. The therapy is expected to be a lifelong solution, with the company behind the development, uniQure, planning further trials in the U.S. and Europe.

Why It's Important?

This development is crucial as Huntington's disease currently has no cure and significantly impacts the lives of those affected, typically leading to a decline in motor and cognitive functions. The potential approval of AMT-130 could provide the first effective treatment for this debilitating condition, offering hope to patients and their families. The therapy's success could also pave the way for similar treatments for other neurodegenerative diseases, potentially transforming the landscape of neurological healthcare. The U.S. Food and Drug Administration (FDA) has already granted the therapy Breakthrough Therapy and Regenerative Medicine Advanced Therapy designations, which could expedite its approval process.

What's Next?

uniQure plans to discuss the trial data with the FDA later this year, aiming to submit a Biologics License Application in early 2026. If approved, this could lead to the therapy's availability in the U.S., followed by applications for approval in the UK and Europe. The results of the trial will be presented at the HD Clinical Research Congress in October, which could further influence the therapy's acceptance and integration into clinical practice.