uniQure and Gen-X Enter into Research Collaboration and License Agreement for the Development of Novel Synthetic Promoters
March 02 2020 - 4:05PM
uniQure N.V. (NASDAQ: QURE), a leading gene therapy company
advancing transformative therapies for patients with severe medical
needs, and Gen-X, a spin-off company from the Netherlands Cancer
Institute, today announced that they have signed an exclusive
research collaboration and license agreement with Gen-X, with an
option for uniQure to acquire Gen-X, to identify novel synthetic
promoters for use in gene therapy products.
Gen-X performs functional annotation of the
non-coding genome using its proprietary Survey of Regulatory
Elements (SuRE) technology. This technology measures the potential
of hundreds of millions of DNA elements for their ability to serve
as promoters or enhancers, driving the gene expression with a range
of unique properties for gene therapy applications. The SuRE
technology is unique among other promoter-identification
technologies in finding non-coding promoter elements throughout the
genome at remote sites where activity is regulated.
“Novel promoters are an important area of
innovation in gene therapy with the potential to enable valuable
functionality and improve treatment options,” stated Jonathan
Garen, chief business officer of uniQure. “With the unique
SuRE methodology developed by Gen-X, we will now leverage our
modular technology platform to potentially identify new promoters
and enhancers that drive gene expression in the right cell-type and
under the right conditions. We are excited about the collaboration,
providing us with the potential of applying this state-of-the-art
technology and increasing the expression and effectiveness of our
next generation gene therapy product candidates.”
During the collaboration, Gen-X will work
exclusively with uniQure to develop promoters for the uniQure
research pipeline. Through the SuRE technology, the Gen-X promoter
library is designed to identify novel promoters to regulate gene
activity and, if needed, conditionally control protein production.
The parties will also explore further development of AAV vector
libraries containing fragments of the non-coding human genome.
Active promoter elements can be directly identified following
administration of these libraries in vivo.
"We are very proud to enter into a partnership
with uniQure, giving us the opportunity to demonstrate the value of
the Gen-X platform in a gene therapy setting,” stated Joris van
Arensbergen, Gen-X founder and CEO. “We believe that combining our
platform with uniQure’s leading expertise in AAV gene therapy will
accelerate the development of advanced gene therapy
candidates.”
Until the expiry of the research term of the
collaboration, uniQure has an option to acquire the issued and
outstanding shares of Gen-X for a pre-agreed price. Financial terms
on the research collaboration and license agreement were not
disclosed.
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 hemophilia B,
hemophilia A, Huntington's disease, Fabry disease, spinocerebellar
ataxia Type 3 and other diseases. www.uniQure.com
About Gen-XGen-X is a
biotechnology company located in Amsterdam, the Netherlands
performing functional annotation of the non-coding genome on an
unprecedented scale using its SuRE technology. The company provides
screening services to gene therapy companies for the identification
of DNA elements that enable controlled transgene expression.
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 generation of new
technology or products, and the payment of associated future
milestones and royalties. Our actual results could differ
materially from those anticipated in these forward-looking
statements for many reasons, including, without limitation, risks
associated with our clinical development activities, clinical
results, collaboration arrangements, 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 Annual Report on Form 10-K filed with
the Securities and Exchange Commission on March 2, 2020. 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.canto@uniQure.com |
e.mulder@uniQure.com |
t.malone@uniQure.com |
uniQure NV (NASDAQ:QURE)
Historical Stock Chart
From Aug 2024 to Sep 2024
uniQure NV (NASDAQ:QURE)
Historical Stock Chart
From Sep 2023 to Sep 2024