UniQure avvio della terapia genetica con ATM-130

UniQure Announces First Two Patients Treated in Phase I/II Clinical Trial of AMT-130 for the Treatment of Huntington’s Disease


Below a brief summary of the official communication from UniQure about the beginning of phase I / II of the gene therapy for Huntington's disease in the United States. The study with the AMT-130 molecule is a randomized, double-blind clinical trial: neither the participants nor the researchers know which of the patients will receive the placebo. On June 19, the first two patients were treated in the United States. Patients were treated through a MRI-guided, convection-enhanced stereotactic neurosurgical delivery directly into the striatum (caudate and putamen), one of them received ATM-130, the other placebo. 

Matt Kapusta, chief executive officer of uniQure, stated: “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”

The Phase I/II clinical trial of AMT-130 for the treatment of Huntington’s disease will explore the safety, tolerability, and efficacy signals in 26 patients with early manifest Huntington’s disease randomized to treatment with AMT-130 or an imitation (sham) surgery. The five-year, multi-center trial consists of a blinded 18-month core study period followed by unblinded long-term follow-up. 

The first two patients will be observed for an initial period of 90 days, followed by a meeting of the Data Safety Monitoring Board (DSMB). The DSMB will review the data on the first two patients and make a determination about continued dosing of the next patients.

Ferdinando Squitieri, LIRH's scientific Director and Head of the Huntington Unit of Casa Sollievo della Sofferenza said: "We hope that this first strategy that involves the use of gene therapy will led, together with other ongoing initiatives, to a new era and unable a positive and substantial change for those suffering from this disease ".

The trial is currently recruiting in five states in the United States: University of California, San Francisco, University of Michigan Department of Neurology, Ohio State University, The University of Texas,  Virginia Commonwealth University VCU School of Medicine, Department of Neurology and University of Washington Medical Center.

Here more information.

Here the official UniQure communication