uniQure Announces Preclinical Proof of Concept for Gene Therapy Approach in Huntington's Disease

uniQure N.V. (NASDAQ:QURE) announced the publication of preclinical data supporting its proprietary Huntington’s disease gene therapy program, AMT-130.
According to the news:

Findings published in the current issue of the peer-reviewed journal Molecular Therapy-Nucleic Acids (www.nature.com/mtna/journal/v5/n3/index.html) provide preclinical proof of concept for uniQure’s AMT-130 program and demonstrate the potential of a one-time administration of AAV5-delivered gene therapy into the central nervous system (CNS) to silence the Huntingtin gene (HTT). An inherited, mutated form of HTT causes Huntington’s disease, a rare, fatal, neurodegenerative disorder that leads to severe physical and cognitive deterioration.

Dan Soland, Chief Executive Officer of uniQure commented:

Dr. Konstantinova and her team have made significant progress in the search for an effective treatment for this cruel neurodegnerative disorder. AMT-130 now represents our third gene therapy product candidate in the CNS area, in addition to AMT-110 in Sanfilippo B and the NIH-sponsored program in Parkinson’s disease. We will continue to leverage our deep experience in the CNS field, as well as our validated manufacturing capabilities and AAV5 technology, to advance AMT-130 towards the clinic.

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