基因疗法成功延缓亨廷顿病发展

Group 1 - The core finding of the study indicates that the gene therapy AMT-130 has successfully slowed disease progression in early-stage Huntington's disease patients by approximately 75% over three years compared to the control group, marking a significant advancement in treatment options [1][2] - AMT-130 utilizes a harmless virus to deliver a microRNA sequence to the affected areas of the brain, effectively "turning off" the defective gene responsible for abnormal protein production [1] - The therapy targets the two brain regions most affected by Huntington's disease, requiring precise injection guided by real-time brain scans, with the entire procedure taking 12 to 18 hours [1] Group 2 - The company plans to submit an application to the U.S. Food and Drug Administration (FDA) early next year, with the potential for the drug to be available on the market by 2027 if approved [3] - Initial results show that the therapy is generally safe, with common side effects including headaches and confusion, which are mostly self-resolving or manageable with steroids [2]