How Genprex Differs From “Traditional” Gene Therapy Approaches
April 12, 2019
Gene therapy is a big and trending topic in the world of medicine and has recently been making headlines as large pharmaceutical companies have been ramping up acquisitions of gene therapy companies. So how exactly does gene therapy work? There are a couple of “traditional” approaches to this generally newer treatment option for rare diseases and cancer. However, Genprex, a clinical-stage gene therapy company developing potentially life-changing technologies for cancer patients based on a unique proprietary technology platform, uses a different approach than those used by other gene therapy companies.
Traditional Gene Therapy Approaches
- Replacement Gene Therapy – this treatment option uses a vector, typically a form of virus, to carry a new copy of a missing or nonworking gene to the control center of the cells where it begins to make the necessary proteins for the body to function properly.1
- Gene Editing – this technique edits, removes or replaces a specific part of a person’s DNA. There are several gene editing techniques, including:
- CRISPR – this technology delivers a substance similar to DNA, called guide RNA, which locates the DNA that needs to be changed. It also delivers a protein, called Cas9, which helps cut out the DNA that needs replaced. The cell then naturally repairs itself and provides a correct set of instructions to for forming healthy cells.2
- ZFN – target genes and change DNA by using engineered molecules of proteins and zinc that bind to DNA. The enzymes this produces bind to specific DNA sequences and cut out and repair the part of the DNA that needs editing.2
- Talens – cuts out specific sequences of DNA through restriction enzymes that bind to DNA.2
- CAR-T Cell Therapy – is a type of cell therapy that uses a person’s own immune cells to fight cancer cells. This therapy works by extracting blood from the patient, removing the immune cells, or T cells, and then adding CAR cells to the T cells. The combined CAR-T cells are then re-introduced back into the patient to stimulate their own immune system to fight off the cancer.3
Genprex’s Gene Therapy Approach
Genprex’s cancer fighting drug candidate for non-small cell lung cancer (NSCLC), Oncoprex™ immunogene therapy, consists of a tumor suppressor gene, which is missing or deficient in many cancers, including lung cancer.
Oncoprex* is encapsulated into a very small vesicle and delivered with cholesterol molecules, a particle the body is familiar with breaking down. Our platform delivery may allow a variety of cancer fighting genes, which are typically missing or deficient in cancer patients, to be combined with cholesterol molecules to help the body fight cancer.
Unlike other gene therapies, our delivery platform does not inject the patient with a virus, and Oncoprex does not edit or modify a patient’s genes.
We are currently conducting clinical trials to evaluate the safety and efficacy of Oncoprex. We currently hold a portfolio of more than 30 issued patents covering our technologies and targeted molecular therapies.
*Oncoprex is currently in development and is not FDA approved.
1. U.S. National Library of Medicine
2. Gupta RM, Musunuru K. Expanding the genetic editing tool kit: ZFNs, TALENs, and CRISPR-Cas9. J Clin Invest. 2014 Oct;124(10):4154-61. doi: 10.1172/JCI72992. Epub 2014 Oct 1. Review. PubMed: 25271723. Free full-text available from PubMed Central: PMC4191047.
3. National Cancer Institute