What's Hot in 2026: Huntington Disease

Could advances in gene therapy finally bring us closer to an effective treatment for HD?

This autumn, the gene therapy company uniQure announced findings from a clinical trial that showed HD patients had a greatly slowed disease progression in patients who received the huntingtin-lowering gene therapy, AMT-130. There are still open questions regarding this trial, and it may be that this type of therapy, in its current form, might not be suitable for all patients. Still, this holds more promise than any existing treatment for HD, and its impact could be truly groundbreaking. This outcome is encouraging for other treatment candidates as well; other clinical trials are ongoing that also have potential. This gives us hope that by leveraging recent scientific advances, we may be closer to an effective treatment for a devastating disease.

-- Taneli Heikkinen, PhD, Senior Scientist, Charles River Laboratories