Gene Therapy, ASOs, and Beyond: What’s Coming for Huntington Disease

Huntington disease (HD) is a progressive, inherited neurodegenerative disorder marked by movement abnormalities, cognitive decline, and psychiatric symptoms. Caused by a CAG repeat expansion in the HTT gene, the resulting toxic buildup of mutant huntingtin protein leads to widespread neuronal damage. Despite advances in genetic testing and diagnosis, there are still no disease-modifying therapies—only symptomatic treatments that offer limited relief and do not slow progression.