uniQure Announces Featured Presentations of New Data on Spinocerebellar Ataxia Type 3 at the 2019 American Academy of Neurology Annual Meeting

uniQure Announces Featured Presentations of New Data on Spinocerebellar Ataxia Type 3 at the 2019 American Academy of Neurology Annual Meeting

Publication date: May 08, 2019

~ Data Show AMT-150 Able to Significantly Lower Mutant Ataxin-3 Protein in SCA3 Disease Model ~ ~ Provides Further Support of Proof-of-concept of Company’s Proprietary miQURE™ Gene Silencing Platform ~ LEXINGTON, Mass. and AMSTERDAM, the Netherlands, May 07, 2019 (GLOBE NEWSWIRE) — uniQure N. V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, will today present preclinical data on its gene therapy candidate, AMT-150, for the treatment of Spinocerebellar Ataxia 3 (SCA3).

uniQure’s AMT-150 incorporates the Company’s proprietary miQURE™ gene silencing technology and comprises an AAV5 vector to deliver a microRNA that is designed to halt ataxia in early manifest SCA3 patients.

We are leveraging our modular and validated technology platform to rapidly advance a pipeline of proprietary and partnered gene therapies to treat patients with hemophilia, Huntington’s disease and other severe genetic diseases.

These forward-looking statements include, but are not limited to, the risk of cessation, delay or lack of success of any of our ongoing or planned clinical studies, our ability to advance our pipeline programs in SCA3 and Huntington’s disease, our ability to move closer to providing potentially transformative therapies to patients and further demonstrate the importance of our industry leading technology platform and AAV manufacturing capabilities, and/or the development and regulatory approval of our product candidates in the United States or in Europe, whether AMT-150 proves to alter the course of this devastating disease after a single administration or at all, whether our miQURE™ technology is able to treat SCA3, Huntington’s disease or a wide range of polyglutamine diseases, and whether we will be able to complete IND-enabling studies.

Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, risks associated with our and our collaborators’ clinical development activities, clinical results, collaboration arrangements, corporate reorganizations and strategic shifts, regulatory oversight, product commercialization and intellectual property claims, as well as the risks, uncertainties and other factors described under the heading “Risk Factors” in uniQure’s Quarterly Report on Form 10-Q filed on April 29, 2019.

Concepts Keywords
AMSTERDAM MiQURE technology
AMT Magnetic resonance imaging
Ataxia Forward-looking statement
Brain Trinucleotide repeat disorder
Cerebrospinal Fluid Machado–Joseph disease
Deventer Neurological disorders
Europe Rare diseases
Genetic Diseases Organ systems
Hemophilia Neurodegenerative disorders
Huntington Autosomal dominant disorders
IND Spinocerebellar ataxia
Intellectual Property Proteins
LEXINGTON Ataxin
Magnetic Resonance Imaging Severe genetic diseases
MicroRNA MiQURE technology
Movement Disorders Halt ataxia
Muscular Atrophy Range polyglutamine diseases
NASDAQ
Netherlands
Neurology
Neurons
Paralysis
Philadelphia
Pipeline
Primates
Progressive
Striatal
Striatum
Tolerability
Vector

Semantics

Type Source Name
gene UNIPROT TNFSF13
gene UNIPROT ANP32B
disease MESH Risk Factors
drug DRUGBANK Spinosad
gene UNIPROT SSRP1
pathway BSID Release
drug DRUGBANK Coenzyme M
disease MESH genetic diseases
disease MESH hemophilia
gene UNIPROT TNIP1
disease MESH ataxia
disease MESH paralysis
disease MESH movement disorders
disease DOID brain degeneration
disease MESH Development
drug DRUGBANK Nonoxynol-9
gene UNIPROT FBN1
gene UNIPROT ATXN3
gene UNIPROT AMT
drug DRUGBANK Alpha-methyltryptamine
disease DOID Spinocerebellar Ataxia Type 3
disease MESH Spinocerebellar Ataxia Type 3

Similar

Leave a Comment

Your email address will not be published. Required fields are marked *