Horizon Discovery Group plc
Exercises Option to Exclusively License
Base Editing Technology from Rutgers, The State University of New
Jersey
Cambridge, UK, 14 January
2020: Horizon Discovery Group plc (LSE: HZD)
("Horizon" or “the Group”), a global leader in the application of
gene editing and gene modulation technologies, announces that it
has exercised its option to exclusively license a novel Base
Editing technology from Rutgers, The State University of New Jersey
(US), for use in therapeutic, diagnostic and service
applications.
In January 2019, Horizon and Rutgers signed an
Option Agreement and a Research Agreement, which granted Horizon an
option to obtain an exclusive license to Rutgers’ proprietary Base
Editing technology for therapeutic, diagnostic and service
applications. Under the terms of the agreement, Horizon and Rutgers
agreed to collaborate to further develop the Base Editing
technology, which included the Group undertaking its own evaluation
and proof-of-concept studies. Horizon has several internal programs
designed to accelerate the clinical uptake of this technology and
make it widely available for therapeutic applications.
Horizon has now exercised its option and Rutgers
has granted it an exclusive license to Rutgers’ proprietary Base
Editing technology. The license includes the right for Horizon to
sublicense the technology to entities seeking to use it for
therapeutic development. Financial terms are confidential and have
not been disclosed.
Base Editing is a novel technology that enables
scientists to make edits to base pairs in DNA by recruiting
specific Base Editing enzymes (deaminases).
Horizon is now seeking early access customers to
assess and shape the development of this Base Editing platform,
which could enable the development of novel therapeutics that rely
on engineering cells either directly in the body (gene therapy), or
externally before transplanting back into the patient (cell
therapy). This novel technology could also be applied in cell
therapies programs that require more effective multi-gene knockouts
(such as CAR-T cells) with an improved safety profile. Customers
will also gain access to Horizon’s expertise in genome engineering
of different cell types, influence over the direction of future
development and access to early technical data from the
platform.
Terry Pizzie, Chief Executive Officer of
Horizon Discovery,
commented:
"Exercising our option for this technology is
potentially transformative for Horizon. Base Editing has the
potential to revolutionise the treatment of genetic diseases. Over
the past year, we completed a detailed evaluation phase of Rutgers’
technology and are excited to now have an exclusive license for use
in therapeutic, diagnostic and service applications.
“There is significant latent demand for access
to Base Editing and we already have significant interest from
potential partners interested in early access to this cutting-edge
technology.”
S. David Kimball, Senior Vice President
for Research and Economic Development, Rutgers University,
added:
“The gene editing technology developed by
Rutgers researchers Dr. Shengkan Jin and Dr. Juan C. Collantes has
the potential to revolutionize how scientists think about their
search for better options and outcomes in the treatment of disease
worldwide. Just as important is our ability, through this
significant partnership with Horizon Discovery, to share our
discoveries and inventions with the scientific community around the
world who are equally committed to improving human
health.
For more scientific detail on Base Editing,
please see below in the notes to editors.
Ends
For further information from Horizon
Discovery Group plc, please contact:
Horizon Discovery Group
plcTerry Pizzie, Chief Executive OfficerJayesh Pankhania,
Chief Financial OfficerJon Davies, Head of Investor RelationsTel:
+44 (0) 1223 655 580
Numis Securities Limited (Broker and
NOMAD)Freddie Barnfield / Tom Ballard / Duncan
MonteithTel: +44 (0) 207 260 1000
Consilium Strategic Communications
(Financial Media and Investor Relations)Mary-Jane Elliott
/ Matthew Neal / Melissa GardinerTel: +44 (0) 20 3709
5700Email: horizon@consilium-comms.com
Zyme Communications (Trade and Regional
Media)Lorna CuddonTel: +44 (0)7787 502
947Email: lorna.cuddon@zymecommunications.com
Westwicke, an ICR Company (US Investor
Relations)Stephanie CarringtonTel. +1
646-277-1282Email: horizondiscovery@icrinc.com
Notes to Editors
About Base Editing
Base Editing is a novel technology platform for
engineering DNA or genes in cells, which allows scientists to make
specific edits to base pairs in DNA or RNA by using an enzyme to
change a single base of DNA into another. Compared with currently
available gene editing methodologies such as CRISPR/Cas9, which
creates "cuts" in the gene that can lead to adverse or negative
effects, this new technology allows for more accurate gene editing
while reducing unintended genomic changes.
Many human diseases have a simple and known
cause, i.e. a single genetic alteration caused through heredity
genetics or by an infectious agent. Examples
include genetic diseases such as sickle cell anaemia and Duchenne
muscular dystrophy, as well as infectious diseases such as AIDS and
hepatitis B. Despite the clear and simple cause, a cure is
understandably difficult because the approved drugs, as well as
most drugs under development, target a disease-associated target or
protein rather than impacting the disease-causing gene itself. This
is largely due to a deficiency in understanding of the etiological
cause of disease as well as the lack of effective DNA-targeting
technology.
CRISPR gene editing technology allows targeted
recognition and modification of specific disease-causing DNA
sequences in the genes of cells. However, first generation CRISPR
technology generates double-strand DNA breaks and often requires
disease tissues or cells to have homologous dependent repair
activity to achieve optimum therapeutic effect. As double strand
DNA breaks are oncogenic in nature and homologous dependent repair
activity is by and large absent in diseased tissues, the first
generation CRISPR technology arguably has major hurdles to overcome
for developing therapeutic vehicles or agents; in particular for
developing in vivo therapeutics since it could generate potentially
oncogenic DNA breaks and usually requires homology-dependent repair
activity which is absent in most disease- affected
organs.
Base Editing has the potential to overcome the
issues above by utilizing a nuclease-deficient CRISPR protein and
an RNA-based recruitment mechanism to guide a non-nuclease DNA
modifying enzyme, such as a cytidine deaminase, to the
disease-causing gene, where the enzyme effectively corrects or
modifies the gene in the disease tissues while minimizing the
generation of the potential oncogenic DNA breaks. This allows more
accurate editing of genes with reduced negative effects due to
unintentional genomic changes.
About Horizon Discovery Group plc
www.horizondiscovery.com
Horizon Discovery Group plc (LSE: HZD)
("Horizon") drives the application of gene editing and gene
modulation within the global life science market – supporting
scientists on the path from research to therapy.
Built upon more than a decade of experience in
the engineering of cell lines, Horizon offers an unmatched
portfolio of tools and services to help scientists gain a greater
understanding of gene function, identify genetic drivers behind
human disease, deliver biotherapeutics, cellular and gene therapies
for precision medicine as well as develop and validate diagnostic
workflows.
Horizon’s solutions enable almost any gene to be
altered, or its function modulated, in human and other mammalian
cell lines.
The Group’s customers include many of the
world’s foremost academic institutes, global pharmaceutical and
biotechnology companies as well as clinical diagnostic
laboratories. Insight into the challenges faced by these
organizations enables Horizon to focus efforts on development of
innovative solutions that not only differentiate the Group’s
offering, but also fuel development of the next wave of precision
medicines.
Horizon is headquartered in Cambridge, UK with
offices in USA and Japan. The Group is listed on the London
Stock Exchange's AIM market under the ticker HZD.
About Rutgers University
Rutgers, The State University of New Jersey, is
a leading national research university and the state of New
Jersey's preeminent, comprehensive public institution of higher
education. Established in 1766, the university is the eighth oldest
higher education institution in the United States. More than 69,000
students and 22,500 full- and part-time faculty and staff learn,
work, and serve the public at Rutgers locations across New Jersey
and around the world.
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