uniQure Announces Publications on AAV Gene Therapy Approach to Treating Amyotrophic Lateral Sclerosis (ALS) and Frontotempora...
February 15 2019 - 7:24AM
uniQure Announces Publications on AAV Gene Therapy Approach to
Treating Amyotrophic Lateral Sclerosis (ALS) and Frontotemporal
Dementia (FTD) in the Journal Molecular Therapy Nucleic Acids
uniQure N.V. (NASDAQ: QURE), a leading gene therapy company
advancing transformative therapies for patients with severe medical
needs, today announced two recent online publications of
preclinical studies showing significant silencing, or knockdown, of
the mutated gene most commonly known to lead to onset of
amyotrophic lateral sclerosis (ALS) and frontotemporal dementia
(FTD), two devastating neurodegenerative diseases. The
proof-of-concept studies were conducted by uniQure scientists and
utilized the Company’s miQURE™ technology, a proprietary,
next-generation gene-silencing platform.
Both studies are published in the scientific
journal, Molecular Therapy Nucleic Acids. The first manuscript
entitled “Artificial microRNAs targeting C9orf72 can reduce
accumulation of the intra-nuclear transcripts in ALS and FTD
patients” describes the design and in vitro characterization of
artificial micro-RNA (miC) that silence the mutated C9orf72 gene.
The second manuscript entitled “Targeting RNA-mediated toxicity in
C9orf72 ALS/FTD by RNAi based gene therapy” reports that an AAV
vector carrying a DNA cassette encoding miC silences the mutated
C9orf72 gene in iPSC-neurons derived from an FTD patient and in an
ALS mouse model that carries the human gene with the C9orf72
mutation. The studies show significant silencing of C9orf72 in
human-derived iPSC neurons, and the mutated C9orf72 was also
reduced in the cell nucleus. AAV5-miC injected into the striatum of
ALS mice reduced mutated C9orf72 in the transduced areas.
“These findings are potentially significant for
the treatment of ALS and FTD patients, and the ability to silence
C9orf72 transcripts in the nucleus may prove to be critical for
therapeutic efficacy of gene therapies for these diseases,” stated
Sander van Deventer, M.D., Ph.D., chief scientific officer of
uniQure. “In ALS, mutated C9orf72 transcipts are confined to the
cell nucleus causing so-called RNA foci, which are toxic clumps of
mutated C9orf72 RNA that sequester critical proteins, resulting in
cellular dysfunction and death. Whereas most miRNA constructs
exclusively target cytoplasmic mRNA, AAV5-miC significantly reduced
formation of RNA foci in the nuclei of neurons from C9orf72
mice.”
“Taken together, these preclinical findings
further support the feasibility of advancing this program through
research and potentially into development of a promising gene
therapy with the potential to alleviate the toxicity caused by the
mutated C9orf72 in ALS and FTD,” he added. “These data illustrate
the potential of our miQURE platform to degrade disease-causing
genes, without off-target toxicity. We are very pleased to have
these data published in a highly relevant journal for the field and
look forward to further exploring this opportunity.”
About miQURE™
miQURE is uniQure’s novel technology platform designed to
degrade disease-causing genes without off-target toxicity, and
induce silencing of the entire target organ through secondary
exosome-mediated delivery. Gene therapy candidates designed with
miQURE incorporate proprietary, therapeutic miRNA constructs that
can be delivered using AAVs to potentially provide long-lasting
activity.
Preclinical studies of miQURE-based gene
therapies have demonstrated several important advantages, including
enhanced tissue-specificity, improved nuclear and cytoplasmic gene
lowering and no off-target effects associated with impact to the
cellular miRNA or mRNA transcriptome. miQURE technology has been
incorporated in AMT-130, an investigational gene therapy for
Huntington’s disease, and is expected to be applied to AMT-150 for
SCA3.
About ALS and FTD
ALS (amyotrophic lateral sclerosis) is a
devastating neurodegenerative disease characterized by progressive
degeneration of the upper and lower motor neurons leading to muscle
atrophy and paralysis. There is no disease-modifying therapy for
ALS and most patients die with respiratory failure within 3-5 years
after the onset of symptoms.
A significant number of ALS patients also
develop FTD (frontotemporal dementia), a presenile form of dementia
characterize by degeneration of neurons in the frontal and temporal
lobes of the brain leading to behavioral changes. The most common
genetic cause of familial and sporadic ALS and FTD is an expanded
GGGGCC (G4C2) repeat in the first intron of the chromosome 9 open
reading frame 72 (C9orf72) gene. Both ALS and FTD often occur at a
relatively young age and have a major impact on the patients and
their families. A mutation in the C9orf72 gene is considered the
most frequent cause of familial ALS and FTD, with some patients
developing both diseases.
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 hemophilia, Huntington’s disease
and other severe genetic diseases. www.uniQure.com
uniQure Forward-Looking
Statements
This press release 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, the development of our
gene therapy product candidates, whether the results of
pre-clinical studies are potentially significant or prove to be
safe or effective in the treatment of ALS and FTD patients, whether
the ability to silence C9orf72 transcripts in the nucleus proves to
be critical for therapeutic efficacy of gene therapies for these
diseases, whether programs involving our miQURE™ technology are
advanced through research or into development or become promising
as gene therapies or can alleviate the toxicity caused by the
mutated C9orf72 in ALS and FTD or in other genes or in other
diseases, delay or lack of success of any of our ongoing or planned
clinical studies and/or development of our product candidates, and
the scope of protection of our proprietary technologies. 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, the success of our pre-clinical
and other research and development efforts, 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 November 6, 2018. 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.
uniQure Contacts:
FOR INVESTORS: |
|
FOR MEDIA: |
|
|
|
Maria E. Cantor |
Eva M. Mulder |
Tom Malone |
Direct: 339-970-7536 |
Direct: +31 20 240 6103 |
Direct: 339-970-7558 |
Mobile: 617-680-9452 |
Mobile: +31 6 52 33 15 79 |
Mobile: 339-223-8541 |
m.cantor@uniQure.com |
e.mulder@uniQure.com |
t.malone@uniQure.com |
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