Agilis Biotherapeutics and Intrexon to Pursue Transformative Therapies for
Rare Genetic Disease
Exclusive Channel Collaboration Focused on DNA-based Therapeutics for Children
and Adults with Friedreich's ataxia
NEW YORK and GERMANTOWN, Md., Dec. 31, 2013
NEW YORK and GERMANTOWN, Md., Dec. 31, 2013 /PRNewswire/ --Agilis
Biotherapeutics, LLC, a synthetic biology-based company focused on rare
genetic diseases, and Intrexon Corporation (NYSE: XON), a leader in synthetic
biology, announced today an Exclusive Channel Collaboration (ECC) to develop
DNA-based therapeutics for Friedreich's ataxia (FRDA), a rare genetic
Agilis and Intrexon expect DNA-based therapeutics to provide the ability to
target underlying disease mechanisms with precision using tightly-controlled
gene therapies for patients with rare inherited diseases such as FRDA.
Current FRDA therapies are primarily focused on supportive care and symptom
relief. There are no FDA-approved treatment options to address the cause of
The technical core of Agilis' novel DNA-based therapeutics will utilize
Intrexon's UltraVector^® platform and RheoSwitch Therapeutic System^® (RTS^®)
to develop gene therapies and genetically-modified cell therapies for treating
FRDA. The ECC's planned approach to target FRDA will employ RTS^®, a
clinically validated inducible gene switch technology that regulates the
expression of therapeutic proteins or bioactive RNA in a dose-dependent
FRDA is an inherited disease caused by a gene mutation that reduces the
expression of frataxin, a protein localized in the "power center" of cells
known as the mitochondria, and results in a physically debilitating,
life-shortening condition. Progression of the disease causes nervous system
damage, problems with movement and early death most often caused by cardiac
malfunction. FRDA is the most common hereditary ataxia with an estimated
5,000 to 10,000 patients in the United States.
The goal of the ECC is to develop DNA-based therapeutics to repair or replace
the "broken" gene in FRDA and enable increased production of the frataxin
protein to alleviate the downstream effects of frataxin deficiency. The
combination of gene correction with additional therapeutic modulators in a
multigenic approach has the potential to create a potent treatment. This
treatment is expected to further improve cardiovascular and neurological
function by addressing the underlying causes of FRDA and associated symptoms.
Agilis also has an option to advance a second undisclosed rare disease
"We are thrilled to be working with Intrexon with the hope of providing
children and adults affected with rare genetic disorders with promising new
treatments," said George S. Zorich, Chief Executive Officer of Agilis
Biotherapeutics. "We believe Agilis is on the forefront of one of the most
promising treatment breakthroughs for Friedreich's ataxia. I am personally
excited to collaborate with the Intrexon team and look forward to developing
new transformative therapies together."
Samuel Broder, M.D., Senior Vice President of Intrexon's Health Sector and
former Director of the National Cancer Institute, said, "FRDA causes heart
failure and progressive neurological deterioration, which in turn cause
suffering and premature deaths. The goal of this collaboration is to harness
Intrexon's proprietary technologies in synthetic DNA, as well as our expertise
in molecular, protein, and cellular engineering, to benefit patients with this
very serious disorder."
Intrexon Founder and Chief Science Officer Thomas Reed, Ph.D., remarked that
unlike some biotechnology companies which are limited to single classes of
therapeutic molecules or gene delivery systems, Intrexon is equipped to pursue
several different therapeutic approaches for treating the complexities
associated with FRDA.
"As an inventor and integrator of technology platforms, we will make use of
our UltraVector® multi-gene engineering, RTS® gene switch, advanced Protein
Engineering, and other platforms to develop therapeutic candidates designed to
treat both the neurological and the cardiovascular pathologies of FRDA. We
are confident that our ability to pursue multiple approaches for treating this
complex disease will significantly increase our probability of success."
About Agilis Biotherapeutics, LLC
Agilis Biotherapeutics, LLC, is a biotechnology company focused on designing
and engineering first-in-class DNA-based therapeutics to improve and save the
lives of patients affected by life-threatening or fatal rare diseases for
which there are no or limited treatment options. We believe that engineered
DNA-based therapeutics provide the ability to target underlying disease
mechanisms with tightly-controlled multigenic modalities leading to
"functional cures" for patients with these diseases.
We invite you to visit Agilis at www.agilisbio.com.
About Intrexon Corporation
Intrexon Corporation (NYSE: XON) is a leader in synthetic biology focused on
collaborating with companies in Health, Food, Energy and the Environment to
create biologically-based products that improve the quality of life and the
health of the planet. Through the company's proprietary UltraVector®
platform, Intrexon provides its partners with industrial-scale design and
development of complex biological systems. The UltraVector® platform delivers
unprecedented control over the quality, function, and performance of living
cells. We call our synthetic biology approach and integrated technologies
Better DNA®, and we invite you to discover more www.DNA.com.
Intrexon, UltraVector, RheoSwitch Therapeutic System, RTS and Better DNA are
trademarks of Intrexon and/or its affiliates. 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 contact:
Chief Executive Officer
Vice President Corporate Communications
Tel: +1 (561) 410-7023
SOURCE Intrexon Corporation
Press spacebar to pause and continue. Press esc to stop.