Gene Therapy Reaches a Tipping Point as Engineered Cell Treatments Break New Ground

UniQure's gene therapy for Huntington's disease slowed disease progression by 75% at three years in a 12-patient study — and four-year data arrives before the end of September. The Dutch biotech has already filed for regulatory approval in both the U.S. and U.K., making AMT-130 one of the most closely watched drugs in biotech right now. For a disease that has zero approved treatments and kills patients in midlife, that number is extraordinary.

From Brain Injections...