uniQure (NASDAQ:QURE) dosed the first two patients in its Phase 1/2 trial evaluating AMT-130 for the treatment of Huntington’s disease (HD).
HD is an inherited neurodegenerative disorder where production and aggregation of an abnormal protein in the brain leads to cognitive decline and loss of muscle coordination. AMT-130 is an adeno-associated virus (AAV) gene therapy that has the potential to lower or knock-down this protein, know as huntingtin, in HD patients.
The five-year trial is designed to explore the safety, tolerability, and efficacy signals in 26 patients with early-manifest HD. Patients will be randomized to receive either a single administration of AMT-130 via MRI-guided, convection-enhanced stereotactic neurosurgical delivery, or a sham surgery.
The first two patients will be observed for 90 days, followed by a meeting of the data safety monitoring board to determine continued dosing of the next patients.
“For years, uniQure has had an unwavering commitment to advance this first-in-human AAV gene therapy for HD into clinical testing, and this moment marks an important milestone for our company now that we have two AAV gene therapy candidates in clinical development,” Matt Kapusta, uniQure’s CEO, said in a statement.
“With the first two patients treated in this trial, we have taken a significant step forward in advancing AMT-130 closer to our goal of developing a therapy that inhibits the production of the mutant huntingtin protein,” he added.
