UniQure Looks For Clear Signal From Huntington’s Disease Gene Therapy

UniQure is awaiting updated data from its potential first-in-class gene therapy, AMT-130, with the most important being a Phase I/II trial in the US monitoring a small group of patients at 12 and 24 months after treatment.

The gene therapy uses an AAV5 vector to transports a micro-RNA into the central nervous system with the goal of silencing the huntingtin gene and blocking production of mutant protein (mHTT), which