- Effective Transduction
Achieved After Readministration of Gene Transfer -
- Preclinical Data Presented
Today at American Society of Gene & Cell Therapy (ASGCT) Annual
Meeting in Washington, D.C. -
- Investor & Analyst
Breakfast and Webcast Friday, May 12 at 7:00 a.m. EDT -
LEXINGTON, Mass. and AMSTERDAM,
the Netherlands, May 11, 2017 (GLOBE NEWSWIRE) -- uniQure
N.V. (NASDAQ:QURE), a leading gene therapy company advancing
transformative therapies for patients with severe medical needs,
today presented data on successful repeated hepatic gene delivery
with uniQure's AAV5 vector following a proprietary immunoadsorption
procedure in non-human primates (NHPs). The results describing
successful sequential transduction with the two reporter transgenes
were presented today in a session at the American Society of Gene
and Cell Therapy (ASGCT) 20th Annual
Meeting in Washington, D.C., and highlight the progress made in
readministration protocols for AAV gene transfers.
"One of the major challenges in
AAV-based gene therapy is the presence of circulating anti-AAV
neutralizing antibodies, which can pre-exist in patients or develop
after a single administration of gene therapy. These neutralizing
antibodies may prevent successful gene transfer in patients,"
stated Valerie Sier-Ferreira, Ph.D., head of immunology at uniQure.
"uniQure has made tremendous progress in optimizing and advancing
readministration protocols that we believe will significantly
impact the application of our gene therapies, potentially enabling
repeated administration and making our gene therapies available to
more patients around the world."
Preclinical Data
Findings
Circulating neutralizing
antibodies (NABs) against AAV vectors can be present in the blood
of patients prior to AAV treatment due to naturally acquired
infections with the wild-type AAV virus. Additionally, anti-AAV NAB
titers are raised to extremely high levels after first
administration of an AAV vector in the course of a gene transfer
treatment. These NABs may prevent successful transduction of the
target cells. As a result, in ongoing clinical trials using AAV
vectors, patients who present detectable levels of anti-AAV
antibodies are excluded from treatment.
In the study conducted by uniQure,
an extracorporeal immune-adsorption (IAd) procedure, similar to
those currently used clinically for the treatment of auto-immune
disorders, was applied to reduce the levels of anti-AAV NABs in
NHPs that previously received an AAV reporter construct
(AAV5-hSEAP). NAB concentrations were decreased by a mean factor of
12, permitting successful readministration of an AAV5 vector
containing a second reporter gene (hFIX). The hFIX concentrations
obtained after re-administration were in the range of the hFIX
levels obtained after primary administration in animals undergoing
IAd, while no hFIX expression was seen in controls who did
not receive IAd prior to readministration. This immune-adsorption
procedure shows promising preclinical outcomes which can
potentially become a successful strategy for readministration of a
gene therapy in a therapeutic regimen.
The poster presented during the
ASGCT Annual Meeting in Washington, D.C., can be accessed by
visiting the Investor relations section of the Company's
website here.
Investor/Analyst Breakfast and Webcast on Friday, May 12,
2017
An investor and analyst breakfast
meeting will be held on Friday, May 12 at 7:00 a.m. EDT. Senior
members of uniQure's research and development team will discuss the
results of this study along with the four additional abstracts that
are being presented at ASGCT. The meeting will be webcast live
along with slides and can be accessed by visiting the investor
relations section of the Company's website
at www.uniQure.com.
Date and
Time: Friday, May 12 at 7:00 am EDT
Location: Omni Shoreham Hotel,
The Congressional Room, 2500 Calvert Street NW, Washington,
D.C.
The Omni Shoreham hotel is located directly across from the
conference venue.
To request attendance at the
meeting, please RSVP to Investors@uniQure.com as space is
limited.
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 cardiovascular
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. Our actual results could differ
materially from those anticipated in these forward-looking
statements for many reasons, including, without limitation, risks
associated with corporate reorganizations and strategic shifts,
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 2016 Annual Report on Form 10-K filed
on March 15, 2017. 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:
Maria E. Cantor
Direct: 339-970-7536
Mobile: 617-680-9452
m.cantor@uniQure.com
Tom Malone
Direct: 339-970-7558
Mobile: 339-223-8541
t.malone@uniQure.com
Eva M. Mulder
Direct: +31 20 240 6103
Mobile: +31 6 52 33 15 79
e.mulder@uniQure.com
This
announcement is distributed by Nasdaq Corporate Solutions on behalf
of Nasdaq Corporate Solutions clients.
The issuer of this announcement warrants that they are solely
responsible for the content, accuracy and originality of the
information contained therein.
Source: uniQure N.V. via Globenewswire
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