DNARx Announces Article in Science Advances about HEDGES™, DNARx’s Proprietary Nonviral Gene Therapy Platform that Overcomes Critical Viral Vector Limitations

SAN FRANCISCO, Dec. 03, 2019 (GLOBE NEWSWIRE) — DNARx, LLC announced today the publication in Science Advances of data demonstrating the capabilities and efficacy of its pioneering HEDGES™ (High-level Extended Duration Gene Expression System) DNA-based, non-viral gene therapy delivery and expression platform. The article appeared on the Science Advances website on Wednesday, November 27, 2019. The data in the article demonstrate significant advantages of HEDGES over viral vector-based approaches to gene therapy.

The published results demonstrate that a single HEDGES administration durably produced FDA-approved protein therapies, including cytokines and monoclonal antibodies at therapeutic levels in mice. These results included:

  • Production of therapeutic human granulocyte colony stimulating factor (hG-CSF, NEUPOGEN®) activity for more than 575 days (>80% of normal mouse lifespan) in fully immune-competent mice
  • Production of extended therapeutic levels of rituximab (Rituxan®), mepolizumab (Nucala®) and an anti-pandemic influenza monoclonal antibody

“The article in Science Advances shows HEDGES’ safety and efficacy as well as HEDGES’ important advantages over viral vector-based gene therapy, such as adeno-associated virus,” said Robert Debs, M.D., CEO of DNARx. These advantages include:

  • HEDGES gene delivery components are not immunogenic, allowing all subjects to receive initial HEDGES treatment, as well as repeated re-treatment
  • HEDGES can be modified to produce either short- or long-term gene expression.
  • HEDGES DNA vectors can accommodate large DNA inserts
    • HEDGES enables expression of large genes
    • HEDGES disease targets are not limited to monogenic diseases
  • HEDGES simultaneously co-expresses multiple different genes
    • HEDGES can address common diseases, such as cancer and heart disease, that require co-expression of multiple genes
  • HEDGES DNA vectors do not detectably integrate into genomic DNA, thereby avoiding possible long-term cancer risks from insertional mutagenesis
  • HEDGES is simpler and more cost-effective to produce than viral vectors, permitting rapid, massive scale up

The potential of HEDGES to prevent and treat infectious disease was recognized by the Defense Advanced Research Projects Agency (DARPA), which earlier this year awarded DNARx a research contract focused on developing a universal preventative and treatment strategy for influenza viral infection.

The completion of the seminal mouse studies described in Science Advances sets the stage for early trials targeting sustained therapeutic protein production in humans.

About DNARx, Inc., and HEDGES™
DNARx is pioneering HEDGES™, an entirely new approach to delivering gene therapeutics that produces human proteins, including monoclonal antibodies, inside patient cells. This revolutionary in-vivo platform has demonstrated the ability to overcome critical drawbacks of currently-employed virus-administered gene therapies. Specifically, HEDGES can be re-dosed because it doesn’t elicit anti-vector adaptive immune responses, HEDGES can deliver gene payloads that are many times the size of current limits, and HEDGES can express human proteins at therapeutic levels for very prolonged periods. HEDGES can also be delivered safely without integrating into the genome, thereby avoiding the risk of the patient developing cancers from insertional mutagenesis that genomic integration can cause.  DNARx has established a broad intellectual property portfolio which covers HEDGES and its uses. The company plans both to develop and commercialize innovative DNA-based drugs and to enter into collaborations with other companies to bring life-saving therapies to patients. DNARx is a privately held company based in San Francisco, California. Visit www.dnarx.net to learn more.


Daniel Dornbusch, DNARx
(415) 497-9933