AMT-130 Gene Therapy Given to 1st Patients in European Trial
The trial is underway in Poland, where it is still open for enrollment, and is expected to expand to other sites in the U.K. and Germany.
“We expect to complete patient enrollment in this European study by the end of the year and to provide safety and target-engagement data from the full 10-patient, low-dose cohort in the U.S. trial in the second quarter of this year,” Ricardo Dolmetsch, PhD, president of research and development at uniQure, said in a press release.
AMT-130 is a gene therapy that uses a short segment of RNA, called microRNA, to prevent the disease-causing mutated huntingtin protein from being made. The therapy fixes to RNA that carries the instructions for the mutated huntingtin protein, marking that RNA for destruction. In this way, AMT-130 targets Huntington’s underlying cause, rather than treating its symptoms.
Both clinical trials are evaluating AMT-130’s safety, tolerability, and efficacy when delivered directly into the brain. A harmless viral vector carrying AMT-130 is administered into the caudate and putamen — two deep brain structures that are responsible for processing information and are affected in people with Huntington’s disease — via a very small catheter. The delivery of AMT-130 into the deep brain structures is monitored using magnetic resonance imaging (MRI).
The European trial (EudraCT 2020-001461-36) will enroll up to 15 patients with early-onset Huntington’s disease, who will be split into low- and high-dose groups. All patients will receive a single administration of AMT-130, after which they will be followed up for an initial six months and then for five years.
“The Interventional Neurotherapy Center (INC) at Mazowiecki Szpital Bródnowski Hospital [in Warsaw, Poland] is the first and only center in Europe currently performing MRI-guided infusions of gene therapies,” said Miroslaw Zabek, MD, PhD, who chairs the center’s department of neurosurgery. “Our team is extremely excited to participate in this important Huntington’s disease scientific research alongside our colleagues in the U.S. and to dose the first patients in the European clinical trial of AMT-130.”
“Since 1995, our center at the Institute of Psychiatry and Neurology (IPiN) has offered genetic testing and clinical care to Polish patients with Huntington’s disease,” said Grzegorz Witkowski, MD, PhD, a principal investigator at the institute. “Our patients have been very interested in the potential for a one-time treatment to stop progression of the disease.”
The trial is currently open for recruitment at IPiN and surgery at INC.