Breakthrough Gene Therapy Treats Huntington’s Disease for First Time

Clinical trials showed that patients kept their independence longer as the disease’s progression slowed dramatically.

A laboratory employee conducts gene therapy experiments in Evry, France, on May 10, 2022. Eric Piermont/AFP

An experimental gene therapy from uniQure slowed the progression of Huntington’s disease by 75 percent over three years, the company said on Sept. 24, raising hopes for the first treatment to alter the course of the fatal brain disorder.

Huntington’s is a rare inherited condition caused by a defective gene that leads to the production of toxic proteins in the brain. Symptoms typically appear in a person’s 30s or 40s and worsen steadily over 10 to 20 years, robbing patients of muscle control, cognitive ability, and independence. There are currently no approved therapies to slow or stop its progression.