Corporate Banner
Satellite Banner
Proteomics
Scientific Community
 
Become a Member | Sign in
Home>News>This Article
  News
Return

Agilis and Intrexon Collaborate on Rare Diseases

Published: Friday, January 03, 2014
Last Updated: Friday, January 03, 2014
Bookmark and Share
Companies announce 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 harnessIntrexon'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.


Further Information

Join For Free

Access to this exclusive content is for Technology Networks Premium members only.

Join Technology Networks Premium for free access to:

  • Exclusive articles
  • Presentations from international conferences
  • Over 3,300+ scientific posters on ePosters
  • More than 4,800+ scientific videos on LabTube
  • 35 community eNewsletters


Sign In



Forgotten your details? Click Here
If you are not a member you can join here

*Please note: By logging into TechnologyNetworks.com you agree to accept the use of cookies. To find out more about the cookies we use and how to delete them, see our privacy policy.


Scientific News
Liquid Biopsies: Miracle Diagnostic or Next New Fad?
Thanks to the development of highly specific gene-amplification and sequencing technologies liquid biopsies access more biomarkers relevant to more cancers than ever before.
New Centre Offers Ultra-Speed Protein Analysis
UW-Madison researchers to establish development centre for next-gen protein measurement technologies.
Protein Nanocages Could Improve Drug Design and Delivery
HHMI scientists have designed and built 10 large protein icosahedra that are similar to viral capsids that carry viral DNA.
Virus Inspired Cell Cargo Ships
Virus-inspired container design may lead to cell cargo ships following construction of ten large, two-component, icosahedral protein complexes.
Protein Reinforces Growth of Damaged Muscles
Biologists have found a protein involved in stem cells that bolsters damaged muscle tissue growth - potential for muscle degeneration treatments.
Structure of Cold Virus Solved
Researchers have identified the structure of an elusive cold virus linked to child asthma and respiratory infections, providing the foundation for treating the virus.
New Protein Model Could Accelerate Drug Development
Stony Brook-led international research team creates ultra-fast approach to model protein interactions.
Researchers Can Control Genes Involved in Cancer
A new way to control the activity of a protein, that is often upregulated in cancer, has been discovered by Moffitt researchers through monoubiquitination mechanism.
Mitochondrial Role in Metastatic Cancer
Researchers have manipulated proteins, sourced from tumour cells, that are essential for maintaining tumour cells and in doing so, have significantly reduced the ability of cancer cells.
Liquid Biopsy Predicts Colon Cancer Recurrence
Scientists have used a genetic test that spots bits of cancer-related DNA circulating in the blood to accurately predict the likelihood of the disease’s return in some — but not all — of a small group of patients with early-stage colon cancer.
Scroll Up
Scroll Down
SELECTBIO

SELECTBIO Market Reports
Go to LabTube
Go to eposters
 
Access to the latest scientific news
Exclusive articles
Upload and share your posters on ePosters
Latest presentations and webinars
View a library of 1,800+ scientific and medical posters
3,300+ scientific and medical posters
A library of 2,500+ scientific videos on LabTube
4,800+ scientific videos
Close
Premium CrownJOIN TECHNOLOGY NETWORKS PREMIUM FOR FREE!