uniQure Announces Successful Readministration of Gene Therapy in Non-Human Primates
May 11 2017 - 7:00AM
– Effective Transduction Achieved After
Readministration of Gene Transfer –
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 EDTLocation: 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
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, 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
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