SIOUX CENTER, Iowa,
April 16, 2018 /PRNewswire/ --
Exemplar Genetics, a wholly owned subsidiary of Intrexon
Corporation (NYSE: XON) committed to enabling the study of
life-threatening human diseases, today announced the U.S. Food
& Drug Administration (FDA) has exercised enforcement
discretion in regard to the ExeGen®ATM MiniSwine model
clearing it for commercial use as a research model. The
ExeGen®ATM MiniSwine is genetically engineered (GE) to
model ataxia telangiectasia (AT), a rare, inherited, predominantly
neurological human disease. Exemplar has previously received
approval for its ExeGen®LDLR MiniSwine model for use in
cardiovascular disease research.
In humans, mutations in the ataxia telangiectasia mutated gene
(ATM) lead to the multisystemic disorder AT. AT is
principally considered a neurodegenerative disease and is
characterized by cerebellar degeneration leading to profound motor
impairment. Additional disease manifestations include fertility
problems, thymic atrophy/hypoplasia, tumor development (mainly
lymphoid organs), and immunological deficiencies. There is
currently no cure for AT and treatment of patients is limited to
supportive care designed to manage disease symptoms.
Following Exemplar's previous approval of its ExeGen®
LDLR MiniSwine model for use in cardiovascular disease research,
the ExeGen® ATM model is the second GE MiniSwine model
reviewed and cleared by the FDA. As a larger animal model,
ExeGen® ATM is designed to enable superior translational
research and better predictive efficacy as scientists seek to
define disease mechanisms and develop novel therapeutics that
address the pathologies of AT. As a result of this approval,
Exemplar's powerful investigational platform can now be offered to
researchers and drug developers.
"The ExeGen® ATM MiniSwine model has allowed us to
study the disease in much greater depth than what was possible
before," commented Jill Weimer,
Ph.D., Senior Director of Therapeutic Development for Sanford
Research. "Through these discoveries we hope to improve our
understanding of the underlying mechanisms of this disease and
develop better methods to treat it."
Small animal models are extensively used in both disease
research and therapeutic development and the current market for
murine models alone exceeds $1
billion. However, the failure of small-animal models
to fully reproduce the human disease condition remains a
significant barrier to defining disease mechanisms and impedes the
development of safe and efficacious therapeutics and diagnostic
tools. Exemplar's GE MiniSwine research models are more
anatomically, physiologically, and genetically similar to humans
than are mice and other small-animal disease models and are
designed to provide improved predictive power to the preclinical
stages of therapeutic development. With failure rates for
therapeutic development programs remaining above 80% and the
estimated average costs of successful development programs above
$1 billion, the need for better and
more predictive research tools remains significant.
"The orphan disease research community remains in desperate need
of better models to advance potential treatments," said
John R. Swart, Ph.D., President and
Chief Executive Officer of Exemplar Genetics. "The
ExeGen® ATM MiniSwine model represents the first of
several models we have developed to enable the advancement of those
therapies. This approval should expand access to our AT model
significantly."
Through its suite of proprietary technologies, Exemplar has
developed a comprehensive pipeline of MiniSwine models to enable
medical research on numerous human health conditions, including
heart disease, cancer, cystic fibrosis, cardiac arrhythmia, and
neuromuscular/neurodegenerative disorders. The ability of
Exemplar's models to more effectively recapitulate human disease
has been well documented through studies led by contract research
organizations as documented in more than 30 peer-reviewed
publications.
About Exemplar Genetics
Exemplar Genetics, a wholly
owned subsidiary of Intrexon Corporation (NYSE: XON), enables
discovery by providing models and services that aid scientists in
the development of next-generation procedures, devices and
therapeutics. Through its innovative models and AAALAC-certified
facilities, Exemplar Genetics assists researchers in making
advances in the discovery of human disease mechanisms, the
optimization of novel diagnostics, and the development of new
treatments. For more information, visit
www.exemplargenetics.com.
Trademarks
ExeGen® is a registered
trademark of Exemplar Genetics. Other names may be trademarks of
their respective owners.
Safe Harbor Statement
Some of the statements made in
this press release are forward-looking statements. These
forward-looking statements are based upon our current expectations
and projections about future events and generally relate to our
plans, objectives and expectations for the development of our
business. Although management believes that the plans and
objectives reflected in or suggested by these forward-looking
statements are reasonable, all forward-looking statements involve
risks and uncertainties and actual future results may be materially
different from the plans, objectives and expectations expressed in
this press release.
For more information regarding Exemplar Genetics,
contact:
General inquiries
contact@exemplargenetics.com
Media inquiries
media@exemplargenetics.com
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SOURCE Intrexon Corporation; Exemplar Genetics