Genprex’s Non-Viral Delivery System Once Again Ahead of Recent Industry Research
December 10, 2019
Researchers at Johns Hopkins Medicine recently published a study in Science Advances that introduces a non-viral delivery system for gene editing. The study, “Carboxylated branched poly(β-amino ester) nanoparticles enable robust cytosolic protein delivery and CRISPR-Cas9 gene editing,” tested the ability of a polymer with positive and negative charges to transmit a protein into cells using CRISPR gene editing. The study found evidence that successful gene editing had occurred.
However, Genprex, a clinical-stage gene therapy company developing potentially life-changing technologies for cancer patients, has already been pioneering the use of non-viral vectors in gene therapy.
Genprex’s Non-Viral Delivery System
Genprex’s non-viral platform technologies are designed to administer cancer fighting genes by encapsulating them into nanoscale hollow spheres called nanovesicles, which are then administered intravenously and preferentially taken up by tumor cells, where they express the necessary proteins that are missing or found in low quantities and that are needed to help kill the cancer cells. The nanovesicles are non-immunogenic, allowing repetitive therapeutic dosing. Genprex’s nanovesicles are also clinically proven to deliver molecular kinase inhibitors effectively.
Designed to Enhance Safety and Efficacy
Genprex has already treated more than 50 patients in Phase I and II clinical trials using its proprietary non-viral delivery system.
A Phase I clinical trial showed that Genprex’s lead drug candidate, Oncoprex™ immunogene therapy, 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.
(Image shows responses in late-stage metastatic lung cancer patient from Phase 1 clinical trial)
Genprex believes this was the first systemic gene therapy clinical trial that used a nanoparticle delivery system for delivering a tumor suppressor gene. Its proprietary nanovesicle gene delivery technology was designed to enhance efficacy and safety.
Non-Viral Delivery Benefits
The company’s nanovesicle non-viral delivery system, which has been used in clinical trials for the treatment of non-small cell lung cancer (NSCLC), is designed to be small enough to cross tight barriers in the lungs but large enough to avoid liver accumulation and clearance.
The nanovesicles are taken up by tumor cells after Oncoprex immunogene treatment at 10 to 25 times the rate at which they are taken up by normal cells.
The TUSC2 gene, which is the active agent in Oncoprex, is encapsulated in a positively charged nanovesicle that binds to actively replicating (and, therefore, negatively charged) cancer cells and then enters the cancer cell through selective endocytosis. The cationic charge of the nanovesicle targets cancer cells, and direct nanovesicle fusion avoids target cell endocytosis.
According to the World Health Organization, lung cancer is the leading cause of cancer deaths worldwide, causing more deaths than breast, colon, kidney, liver, prostate or skin cancers, and lung cancer is one of the most common types of cancer. Each year, there are more than 2 million new lung cancer cases and 1.7 million deaths from lung cancer worldwide. According to the American Cancer Society’s 2019 report, each year in the United States there are more than 228,000 new cases of lung cancer and more than 142,000 deaths from lung cancer.
Genprex’s innovative science has been leading the field of gene therapy, and its non-viral delivery system could potentially unleash the power of delivering effective cancer treatments.
*Oncoprex is currently in development and is not FDA approved