uniQure Announces Presentations at the Annual Meeting of The European Society of Gene and Cell Therapy (ESGCT)
October 19 2021 - 7:05AM
uniQure Announces Presentations at the Annual Meeting of The
European Society of Gene and Cell Therapy (ESGCT)
uniQure N.V. (NASDAQ: QURE), a leading gene therapy company
advancing transformative therapies for patients with severe medical
needs, today announced that four data presentations, including one
oral presentation, will be delivered at the European Society of
Gene and Cell Therapy (ESGCT) 28th Annual Meeting taking place
virtually today through October 22, 2021.
Featured in the oral presentation are
preclinical data of AMT-191, uniQure’s gene-therapy candidate for
the treatment of Fabry disease which is advancing into IND-enabling
studies. AMT-191 is a one-time administered AAV5 gene therapy
incorporating an α-galactosidase A (GLA) transgene. New preclinical
data confirms high efficiency and cross correction of AMT-191 in a
Fabry mouse model, with increased GLA-activity in the liver,
kidney, heart, and brain and normalized (lyso-)Gb3 levels in main
target organs. In addition, a single administration of AMT-191 led
to phenotype improvement of nociception or a faster reaction time
to physical stimulus.
These new data build on earlier preclinical
studies comparing multiple product candidates, including constructs
incorporating a modified alpha-N-acetylgalactosaminidase transgene
(modNAGA) where AMT-191 demonstrated robust and sustained increases
in GLA activity and subsequent functional improvement.
Moreover, pre-clinical studies in non-human primates with AMT-191
demonstrated robust and sustained increases in GLA activity in
multiple organs.
“We are enthusiastic about sharing these data on
our Fabry program with the scientific community, and we plan to
accelerate the preclinical work necessary to file an IND for
AMT-191 by 2023,” stated Ricardo Dolmetsch, Ph.D., president of
research and development at uniQure. “We believe that this
preclinical data on AMT-191 showing high efficiency and cross
correction in a mouse model differentiates this program from other
current investigational gene therapy programs in Fabry disease. We
look forward to accelerating this program closer to IND filing and
the clinic.”
Specific details on uniQure’s preclinical data
presentations at ESGCT include:
- Oral Presentation
Title: Pre-clinical proof of concept of an AAV5-GLA gene
therapy for Fabry disease results in cross-correction in GLA-KO
mice and non-human primates in target organsDate and
Time: Wednesday, October 20, 2021, 4:15 - 4:30 p.m. CEST
(10:15 – 10:30 a.m. ET)Location: Session 3a: Liver
& Metabolic Disease Studio 1Summary: AMT191,
the company’s investigational AAV5 gene therapy for Fabry disease,
containing a proprietary promoter and the GLA enzyme, reduces GB3
accumulation in mouse liver, kidney, heart and brain.
- Title: Dose
dependent lowering of alpha-synuclein and rescue of motor phenotype
by miRNA-based AAV gene therapyDate and Time:
Tuesday, October 19, 2021, 8:00 a.m. CEST (2:00 a.m.
ET)Location: Poster P073Summary:
AMT-230, the company’s investigational AAV gene therapy for
Parkinson’s Disease, reduces alpha synuclein in dopaminergic
neurons and rescues motor phenotypes in a genetic model of
Parkinson’s Disease.
- Title: Efficacy of
C9orf72 ALS gene therapy using miQURE® and widespread distribution
in cortical and spinal regions in non-human primatesDate
and Time: Tuesday, October 19, 2021, 8:00 a.m. CEST (2:00
a.m. ET)Location: Poster
P052Summary: AMT-161, the Company’s
investigational AAV gene therapy for ALS, reduces the expression of
c9ORF72 in human iPSC derived neurons, reduces C9ORF72-containing
RNA foci in a model of the disease and is distributed to relevant
regions of the spinal cord and brain when expressed in non-human
primates.
- Title: An AAV9
vector expressing engineered miRNA targeting knockdown of
GluK2-containing kainate receptors as a novel gene therapy approach
for treating intractable temporal lobe epilepsyDate and
Time: Tuesday, October 19, 2021, 8:00 a.m. CEST (2:00 a.m.
ET)Location: Poster P074Summary:
AMT-260, the Company’s investigational AAV gene therapy for
temporal lobe epilepsy, efficiently reduces the expression of GluK2
in cortical neurons, reduces epileptiform activity and
hyperlocomotion in a preclinical model of epilepsy and blocks
epileptiform discharges in organotypic slices from patients with
TLE.
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 gene therapies to treat patients with severe genetic
diseases of the central nervous system (CNS) and liver, including
clinical programs in hemophilia B and Huntington's disease and
preclinical candidates in Fabry disease, spinocerebellar ataxia
Type 3, temporal lobe epilepsy, Alzheimer’s, Parkinson’s, and ALS.
www.uniQure.com
unique Forward-Looking
StatementsThis 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, whether we will be able
to accelerate the preclinical for AMT-191 or file an IND for
AMT-191 by early 2023. Our actual results could differ materially
from those anticipated in these forward-looking statements for many
reasons, including, without limitation, risks associated with the
COVID-19 pandemic, our and our collaborators' development
activities, collaboration arrangements, strategic shifts,
regulatory oversight, 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 July 26, 2021. 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 |
Chiara
Russo |
Tom Malone |
Direct: 339-970-7536 |
Direct: 617-306-9137 |
Direct: 339-970-7558 |
m.cantor@uniQure.com |
c.russo@uniQure.com |
t.malone@uniQure.com |
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