uniQure Announces Preclinical Proof of Concept for Gene Therapy Approach in Huntington's Disease
March 22 2016 - 11:05AM
uniQure N.V. (NASDAQ: QURE), a leader in human gene therapy, today
announced the publication of preclinical data supporting its
proprietary Huntington's disease gene therapy program, AMT-130.
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.
The paper, titled "Design, Characterization, and Lead Selection
of Therapeutic miRNAs Targeting Huntingtin for Development of Gene
Therapy for Huntington's Disease", was authored by a research team
led by Pavlina Konstantinova, Ph.D., Director of Emerging
Technologies at uniQure under the direction of Chief Scientific
Officer Harald Petry, Ph.D. The publication describes multiple
approaches to silencing HTT using expression cassette-optimized
artificial microRNAs (miHTTs). Several miHTT scaffolds were
incorporated in an AAV5 vector using uniQure's established
baculovirus-based manufacturing platform and administered to a
humanized mouse model. The data demonstrate strong silencing of
mutant HTT and total HTT silencing in vitro and in vivo.
Furthermore, it was shown that HTT knock-down efficiency could be
increased to 80% by using optimized miHTT scaffolds. The data
published today were in part presented at the 11th Annual CHDI
Huntington's Disease Therapeutics Conference on February 24, 2016
by Dr. Konstantinova.
Based on these results, uniQure has initiated further studies of
AMT-130 to support the filing of an investigative new drug
application with the FDA.
"Huntington's disease devastates families and there is currently
no effective disease-modifying treatment," commented Charles W.
Richard, M.D., Ph.D., Senior Vice President, Research and
Development, Neuroscience at uniQure. "We are excited by the
results of this study, and believe this degree of knock-down of
mutant Huntingtin protein, if duplicated in our ongoing non-human
primate safety toxicology studies and future human clinical trials,
could significantly alter the course of the disease."
"Dr. Konstantinova and her team have made significant progress
in the search for an effective treatment for this cruel
neurodegnerative disorder," said Dan Soland, Chief Executive
Officer of uniQure. "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."
About Huntington's Disease
Huntington's disease is a severe genetic neurodegenerative
disorder causing loss of muscle coordination, behavioral
abnormalities and cognitive decline, resulting in complete physical
and mental deterioration over a 12-15 year period of time. The
disease is caused by an autosomal dominant mutation, a
cytosine-adenine-guanine (CAG) expansion, in the first exon of the
Huntingtin gene leading to a non-functional, aggregation prone
mutated protein. Despite the clear etiology, there are no therapies
available to treat the disease, delay onset or slow progression of
a patient's decline.
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 and partnered gene therapies to treat
patients with CNS, liver/metabolic and cardiovascular diseases.
www.uniQure.com
uniQure Forward-Looking Statement
This presentation 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, statements regarding the future
development of our programs in Huntington's disease, Parkinson's
disease and Sanfilippo B, and the progress of any of our ongoing or
planned clinical studies and/or development of our product
candidates. Our actual results could differ materially from those
anticipated in these forward-looking statements for many reasons,
including, without limitation, risks associated with collaboration
arrangements, our and our collaborators' clinical development
activities, 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 2014 Annual Report on Form 20-F filed with the Securities
and Exchange Commission on April 7, 2015. Given these risks,
uncertainties and other factors, you should not place undue
reliance on these forward-looking statements, and we assume no
obligation to update these forward-looking statements, even if new
information becomes available in the future.
CONTACT: uniQure:
Eva Mulder
Direct: +31 20 240 6103
Main: +31 20 240 6000
e.mulder@uniQure.com
Media inquiries:
Gretchen Schweitzer
MacDougall Biomedical Communications
Direct: +49 172 861 8540
Main: +49 89 2424 3494 or +1 781 235 3060
gschweitzer@macbiocom.com
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