Genprex is Pioneering the Use of Non-Viral Vectors in Gene Therapy
December 17, 2019
Genprex, a clinical-stage gene therapy company developing potentially life-changing technologies for cancer patients, is once again leading the way in gene therapy and raising the standard in cancer treatment based upon its unique, proprietary technology platform that includes its non-viral vector nanoparticle delivery system.
In gene therapy for cancer treatment, vectors are the vehicles that deliver a new copy of a missing or nonworking gene to the control center of the cancer cells.1 Most gene therapy research has been focused on using viral delivery systems to deliver genes to cancer cells2, and today most approved gene therapies for non-blood cell therapies use a viral vector to deliver the gene to the patient.3
But what are the downsides to using a viral vector? There are a few.
First, viral vectors use a virus to complete the gene transfer to the patient. These viruses include lentiviruses, retroviruses, oncoretroviruses, adenoviruses, herpes-simplex viruses, and adeno-associated viruses. Using a virus to deliver a gene to a patient has its risks, and there have been instances where patients have had severe adverse reactions to the use of viral vectors.4
Second, viral vector manufacturing is associated with high production costs, and they are difficult to scale.4Production of recombinant viral vectors is seen as a complex process that faces many challenges for commercialization.5
Third, viral vectors have been shown to have high immunogenicity responses for specific therapeutic applications and a short-lived transgene expression.4As viruses evolve, so too does the body’s immune system, and many times a patient’s immune system fights off the virus in the viral vector because it perceives it as an invading threat.6
New research is being done to test the safety of non-viral vectors in gene therapy. Earlier this year, researchers in Australia studied biomolecule-metal-organic framework (MOF) in zeolitic imidazolate framework-8 (ZIF-8) and found it to be a viable vehicle for intracellular transfection and gene delivery.
These researchers believe that a non-viral delivery system would be safer for patients and could speed up the production time while reducing the production expense. This would mean patients could get a safer therapy for less.
However, Genprex has already been leading the charge to tackle cancer treatment with a non-viral delivery system. Genprex has already treated patients in clinical trials using its proprietary non-viral delivery system.
Genprex’s Non-Viral Nanoparticle Delivery System
Genprex’s platform technologies are designed to administer cancer fighting genes by encapsulating them into nanoscale hollow spheres called nanovesicles, which are then administered intravenously and taken up by tumor cells where they express proteins that are missing or found in low quantities. The nanovesicles are non-immunogenic, allowing repetitive therapeutic dosing. Genprex’s nanovesicles are also clinically proven to deliver molecular kinase inhibitors effectively.
A Phase I clinical trial showed that Genprex’s lead drug candidate, Oncoprex, which is delivered through its nanovesicle non-viral delivery system, was proven to selectively and preferentially target primary and metastatic tumor cells, resulting in clinically significant anticancer activity.
Clinical trials have also demonstrated that the delivery system used in Oncoprex is well tolerated in humans and can safely deliver high therapeutic doses.
Genprex’s efforts to tackle cancer with a non-viral delivery system are nothing short of pioneering, and the Company is well positioned to unleash the full potential of gene therapy through its innovative science.
*Oncoprex is currently in development and is not FDA approved.
- How does gene therapy work?. [online] Genetics Home Reference. Available at: https://ghr.nlm.nih.gov/primer/therapy/procedures [Accessed 7 Aug. 2019].
- Non-viral gene therapy to speed up cancer research. [online] Scienmag: Latest Science and Health News. Available at: https://scienmag.com/non-viral-gene-therapy-to-speed-up-cancer-research/[Accessed 26 Sept. 2019].
- Approved Cellular and Gene Therapy Products. [online] U.S. Food and Drug Administration. Available at: https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/approved-cellular-and-gene-therapy-products[Accessed 26 Sept. 2019].
- Thomas CE, et al. Progress and problems with the use of viral vectors for gene therapy. Nature Reviews Genetics. 2003, May 1. doi: 10.1038/nrg1066.
- AAV VECTOR MANUFACTURING – Challenges & Opportunities in the Manufacturing of AAV Vectors Used in the Delivery of Gene Therapy Treatments. [online] Drug Development and Delivery. Available at: https://drug-dev.com/aav-vector-manufacturing-challenges-opportunities-in-the-manufacturing-of-aav-vectors-used-in-the-delivery-of-gene-therapy-treatments/[Accessed 26 Sept. 2019].
- Nayak S, Herzog RW. Progress and prospects: immune responses to viral vectors [published correction appears in Gene Ther. 2010 Feb;17(2):294]. Gene Ther. 2010;17(3):295–304. doi:10.1038/gt.2009.148