Experimental Gene Therapy AMT-130 Slows Huntington’s Disease Progression by 75% in Pivotal Phase 1/2 Study

 
An experimental gene therapy has been found to slow the progression of Huntington’s disease in a “pivotal” Phase 1/2 study, according to the drugmaker, marking a major step toward a potential first genetic treatment for the condition. The Amsterdam-based company announced yesterday that patients who received a high dose of its AMT-130 therapy for Huntington’s disease saw disease progression slow by 75 per cent after 36 months. The full study results have not yet been published in a peer-reviewed journal.
 
 
Treatment uses a harmless virus to deliver microRNA into the brain. This molecule blocks the faulty genetic instructions that cause the harmful buildup of the mutant huntingtin protein, the key driver of the disease.
 
 
Trial results show promise, marking a potential breakthrough not only for Huntington’s but also for other neurodegenerative diseases like Parkinson’s and Alzheimer’s.