A novel gene therapy has been found to significantly slow the progression of Huntington’s disease , offering new hope to sufferers of the incurable disorder that destroys brain cells over time.

Drugmaker uniQure reported the results of its small but “groundbreaking” Phase I/II study this week, finding that a high dose of its AMT-130 therapy slowed disease progression by 75% after 36 months.

“These findings reinforce our conviction that AMT-130 has the potential to fundamentally transform the treatment landscape for Huntington’s disease, while also providing important evidence supporting one-time, precision-delivered gene therapies for the treatment of neurological disorders,” Dr. Walid Abi-Saab , chief medical officer of uniQure, said as part of Wednesday’s announcement.

Huntingto

See Full Page