FDA Clears Gene Therapy for Huntington Disease Assessment

An Investigational New Drug application was approved to clinically assess AMT-130 as the first potential drug marketed for the neurodegenerative disease.


The US Food and Drug Administration (FDA) has granted Investigational New Drug (IND) application clearance to a gene therapy for Huntington Disease (HD). The clearance indicates that gene therapy AMT-130 can now be reviewed in patients with the rare neurological disorder in clinical settings.

AMT-130, from uniQure NV, will now be observed in a planned dose-escalating, randomized, controlled phase 1/2 study that will assess its safety, tolerability, and efficacy as a one-time treatment in patients with HD. Several US-based clinical sites designated for the trial are anticipated to be opened, and the first patients should be dosed in the second half of this year.

A recombinant AAV5 vector carrying a DNA cassette encoding a microRNA, AMT-130 non-selectively lowers or knocks down huntingtin protein in human patients. It could seek to become the first therapy marketed to treat, delay onset, or even slow progression of the rare inherited disorder which causes patients to lose coordination of their muscles, behavioral normality, and cognitive function.

HD is caused by autosomal dominant mutation that leads to mutated protein production and aggregation in the brain. Despite this etiology being well-established, beneficial therapeutic options elude investigators.

This is what makes the IND clearance for AMT-130 a significant milestone in the field, Matt Kapusta, chief executive officer at uniQure, said in a statement.

“AMT-130 also represents the first clinical-stage AAV-based therapy specifically designed to silence an abnormal gene in the brain with a single administration, and we believe our proprietary miQURE™ gene silencing platform has the potential to be applied to many other diseases, such as spinocerebellar ataxia type 3 (SCA3),” he said. “This achievement is a major milestone for uniQure’s research organization, who have dedicated years of effort with the hope we can one day offer treatment for the many patients waiting generations for an effective therapy.”

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