uniQure Presents Multiple New Preclinical Data on AMT-130 at the CHDI’s 15th Annual Huntington’s Disease Therapeutics Conference

uniQure Presents Multiple New Preclinical Data on AMT-130 at the CHDI’s 15th Annual Huntington’s Disease Therapeutics Conference

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.

Concepts Keywords
AMSTERDAM Spectroscopy
AMT Gene Therapy
Autosomal Dominant Extracellular vesicle
Biomarker Huntingtons
Brain Forward-looking statement
California Huntington’s disease
Cognitive Medical signs
Etiology RTT
Exon Cell biology
Extracellular Vesicles Biotechnology
Fabry Disease Biomarkers
Gene Medicine
Hemophilia Branches of biology
Huntingtin Neurodegenerative disorder
Huntington Onset slow diseases
Intellectual Property
LEXINGTON
Muscle
NASDAQ
Netherlands
Neurodegenerative Disorder
Palm Springs
Pipeline
Resonance
Spectroscopy
Spinocerebellar Ataxia
Vector

Semantics

Type Source Name
disease MESH Risk Factors
disease MESH spinocerebellar ataxia Type 3
disease MESH Fabry disease
disease MESH hemophilia A
disease MESH hemophilia B
disease MESH neurodegenerative disorder
disease MESH abnormalities
disease MESH cognitive decline
drug DRUGBANK Tropicamide
disease MESH development
drug DRUGBANK Nonoxynol-9
drug DRUGBANK Alpha-methyltryptamine
pathway KEGG Huntington disease
disease MESH Huntington Disease

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