“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
“The Interventional Neurotherapy Center (INC) at Mazowiecki Szpital Bródnowski Hospital is the first and only center in
“Since 1995, our center at the
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
About AMT-130
AMT-130 is uniQure’s first central nervous system (CNS) focused gene therapy product consisting of an AAV5 vector carrying an artificial micro-RNA specifically tailored to silence the huntingtin gene, leveraging our proprietary miQURE® silencing technology. The therapeutic goal is to inhibit the production of the mutant protein (mHTT). Using AAV vectors to deliver micro-RNAs directly to the brain for non-selective knockdown of the huntingtin gene represents a highly innovative and promising approach to treating Huntington’s disease.
About Huntington’s Disease
Huntington’s disease is a rare, inherited neurodegenerative disorder that leads to motor symptoms including chorea, and behavioral abnormalities and cognitive decline resulting in progressive physical and mental deterioration. The disease is an autosomal dominant condition with a disease-causing CAG repeat expansion in the first exon of the huntingtin gene that leads to the production and aggregation of abnormal protein in the brain. Despite the clear etiology of Huntington’s disease, there are no currently approved therapies to delay the onset or to slow the disease’s progression.
About uniQure
uniQure is delivering on the promise of gene therapy – single treatments with potentially curative results. We are leveraging our modular and validated technology platform to rapidly advance a pipeline of proprietary gene therapies to treat patients with hemophilia B,
uniQure Forward-Looking Statements
This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as "anticipate," "believe," "could," "estimate," "expect," "goal," "intend," "look forward to", "may," "plan," "potential," "predict," "project," "should," "will," "would" and similar expressions. Forward-looking statements are based on management's beliefs and assumptions and on information available to management only as of the date of this press release. These forward-looking statements include, but are not limited to, whether we will complete patient enrollment in the European study by the end of this year or ever, whether we will be able to provide safety and target-engagement data from the low-dose cohort in the
uniQure Contacts: | ||||
FOR INVESTORS: | FOR MEDIA: | |||
Maria E. Cantor Direct: 339-970-7536 Mobile: 617-680-9452 m.cantor@uniQure.com | Chiara Russo Direct: 617-306-9137 Mobile: 617-306-9137 c.russo@uniQure.com | Direct: 339-970-7558 Mobile: 339-223-8541 t.malone@uniQure.com |
Source: uniQure Inc.
2022 GlobeNewswire, Inc., source