Publication date: Feb 28, 2020
~ Up to Two Years of Follow-up in Large Transgenic Huntington’s Disease Model Demonstrates Stable mHTT Protein Lowering ~ ~ Additional Data Demonstrates the Potential of MRS as Imaging Biomarker for Huntington’s Disease Gene-Therapy Studies ~ LEXINGTON, Mass. and AMSTERDAM, the Netherlands, Feb. 27, 2020 (GLOBE NEWSWIRE) — (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced the presentation of multiple new preclinical data on AMT-130, its investigational AAV gene therapy for the treatment of (HD), at the 15th Annual CHDI Huntington’s disease Therapeutics Conference in Palm Springs, California.
-Our data presentations at CHDI illustrate the increasing potential of AMT-130 to target the highly toxic exon 1 protein fragment, achieve broad vector biodistribution across several animal species and show meaningful activity using the presence of extracellular vesicles as a potential biomarker,” stated , M. D., Ph. D., executive vice president, research & product development of uniQure.
Important findings across several preclinical studies presented at the conference include the following: Translatable Biomarkers in Gene Therapy for Huntington Disease: Learnings from Pre-clinical Studies Exploring the Effects of Intrastriatal AAV5-miHTT Lowering Therapy on Neuronal Function, MRS Signal and Mutant Huntingtin Levels in the Q175FDN Mouse Model of Huntington’s disease The uniQure data presentations featured at CHDI are available on the investor page of the Company’s website, About Huntington’s Disease Huntington’s disease is a rare, inherited neurodegenerative disorder that leads to loss of muscle coordination, behavioral abnormalities and cognitive decline, resulting in complete physical and mental deterioration.
These forward-looking statements include, but are not limited to, whether AMT-130 will target the highly toxic exon 1 protein fragment, achieve broad vector biodistribution across several animal species or show meaningful activity using the presence of extracellular vesicles as a potential biomarker, and whether magnetic resonance spectroscopy will be an important imaging biomarker to measure the restoration of target tissue.
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