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First Patients dosing in European Open-Label AMT-130 Gene Therapy trial for Huntington's

uniQure announced the dosing of the first two patients in its European open-label Phase Ib/II clinical trial of AMT-130, a potential one-time gene-therapy approach for the treatment of Huntington’s disease. The clinical trial is taking place at several sites in Poland, the United Kingdom and Germany.

On the last February 2, 2022 uniQure sent out a press release with new updates on their gene therapy for Huntignton Disease.

“We are very pleased to expand the clinical development of AMT-130 and to build on our ongoing experience in the Phase I/II clinical trial in the United States,” stated Ricardo Dolmetsch, Ph.D., president of research and development at uniQure. “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.”

The European Phase Ib/II clinical trial of AMT-130 for the treatment of Huntington’s disease will explore the safety, proof of concept, and dosing in 15 total patients with early manifest Huntington’s disease split into a five person, low-dose open-label cohort, followed by a nine patient, higher-dose open-label cohort. All patients will be dosed with AMT-130.

The multi-center study consists of an initial 6-month post-treatment study period followed by long-term follow-up for five years. Patients will receive a single administration of AMT-130 through MRI-guided, convection-enhanced stereotactic neurosurgical delivery directly into the striatum (caudate and putamen). The study is currently open for recruitment at IPiN and surgery at INC in Poland and is expected to expand to referral and surgical sites in the United Kingdom and Germany.


Additional details on (EudraCT 2020-001461-36).

Learn more on this study here:  QUI

Read the previous updates here QUI