Navigation Links
Agilis Biotherapeutics and Intrexon to Pursue Transformative Therapies for Rare Genetic Disease

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 neurodegenerative disease.

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 FRDA.

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 fashion.

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 indication.

"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  

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

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:

Agilis Biotherapeutics
George Zorich
Chief Executive Officer

Intrexon Corporation
Peter McLaughlin
Vice President Corporate Communications
Tel:      +1 (561) 410-7023

SOURCE Intrexon Corporation
Copyright©2012 PR Newswire.
All rights reserved

Related biology technology :

1. Healthpoint Biotherapeutics to Present at 30th Annual J.P. Morgan Healthcare Conference
2. Oxford BioTherapeutics Appoints C. Glenn Begley as a Senior Clinical Advisor and Non-executive Director
3. Healthpoint Biotherapeutics To Create State-of-the-Art Cell Sciences Manufacturing Facility
4. Northwest Biotherapeutics Appoints Pharma Industry Veteran As CEO Of German Subsidiary
5. Smith & Nephew Announces Agreement To Acquire Assets Of Healthpoint Biotherapeutics
6. Genedata Expressionist for Mass Spectrometry Showcased at 2013 Biotherapeutics Analytical Summit
7. Oxford BioTherapeutics and Boehringer Ingelheim Enter Collaboration to Discover Novel Antibody Targets in Cancer
8. Acquisitions, Project Announcements & Trend Analysis News for Oxygen Biotherapeutics, Glu Mobile, Cisco Systems, Barrick Gold, CVS Caremark
9. Northwest Biotherapeutics Announces Pricing Of Public Offering Of Common Stock And Warrants
10. Northwest Biotherapeutics to Present at the Oppenheimer 24th Annual Healthcare Conference On December 10, 2013
11. Synthetic Biologics and Intrexon Corporation Enter Worldwide Exclusive Collaboration for Infectious Diseases
Post Your Comments:
(Date:11/25/2015)... -- PharmAthene, Inc. (NYSE MKT: PIP) announced  today that its ... (Rights Plan) in an effort to preserve the value ... 382 of the Internal Revenue Code (Code). ... its NOLs could be substantially limited if the Company ... of the Code. In general, an ownership change occurs ...
(Date:11/25/2015)... 2015 Studies reveal the differences ... and pave the way for more effective treatment for one ...   --> --> ... problems in cats, yet relatively little was understood about the ... have been conducted by researchers from the WALTHAM Centre for ...
(Date:11/25/2015)... , Nov. 25, 2015  Neurocrine Biosciences, Inc. ... Gorman , President and CEO of Neurocrine Biosciences, will ... Conference in New York . ... visit the website approximately 5 minutes prior to the ... replay of the presentation will be available on the ...
(Date:11/25/2015)... ... November 25, 2015 , ... A long-standing ... Aerospace Professionals (OPBAP) has been formalized with the signing of a Memorandum of ... with OPBAP leaders Capt. Karl Minter and Capt. Albert Glenn Tuesday, November 24, ...
Breaking Biology Technology:
(Date:11/12/2015)... , Nov. 12, 2015  Arxspan has entered ... MIT and Harvard for use of its ArxLab ... management tools. The partnership will support the institute,s ... and chemical research information internally and with external ... used for managing the Institute,s electronic laboratory notebook, ...
(Date:11/9/2015)... , Nov. 09, 2015 ... the addition of the "Global Law ... their offering. --> ) ... "Global Law Enforcement Biometrics Market 2015-2019" ... Research and Markets ( ) has ...
(Date:10/29/2015)... Oct. 29, 2015  The J. Craig Venter Institute ... "DNA Synthesis and Biosecurity: Lessons Learned and Options for ... Health and Human Services guidance for synthetic biology providers ... --> --> Synthetic ... the potential to pose unique biosecurity threats. It now ...
Breaking Biology News(10 mins):