21st Century Gene Therapies

Our technologies are designed to administer disease-fighting genes to provide new therapies for large patient populations with cancer and diabetes who currently have limited treatment options. Our teams work closely with world-class researchers, institutions and industry collaborators to expand our pipeline of gene therapies and provide a wider range of novel treatment approaches for patients with cancer and diabetes.

Systemic Gene Therapy Platform for Cancer

Genprex’s oncology drug development program utilizes its unique, proprietary, non-viral ONCOPREX® Nanoparticle Delivery System, which we believe is the first systemic gene therapy delivery platform used for cancer in humans. Using this system, we encapsulate plasmids that express tumor suppressor genes within lipid nanoparticles and intravenously administer the encapsulated plasmids which are taken up by the tumor cells, after which the tumor suppressor genes express proteins that are missing or found in low quantities in the tumor cells. 

REQORSA™ Immunogene Therapy

Our lead product candidate, REQORSA®  immunogene therapy drug (quaratusugene ozeplasmid) for non-small cell lung cancer (NSCLC) and small cell lung cancer (SCLC), uses our unique, proprietary ONCOPREX® nanoparticle delivery system, a systemic gene therapy platform for cancer. The active ingredient in REQORSA is the TUSC2 gene, a tumor suppressor gene. In 2020, the FDA granted Fast Track Designation for REQORSA in combination with AstraZeneca’s Tagrisso® (osimertinib) in late-stage NSCLC patients with EFGR mutations whose tumors progressed after treatment with Tagrisso. In 2021, the FDA granted Fast Track Designation for REQORSA in combination with Merck & Co’s Keytruda® (pembrolizumab) in late-stage NSCLC patients whose disease progressed after treatment with Keytruda.

Diabetes Gene Therapy

GPX-002, a gene therapy for diabetes, is the most recent addition of our licensed technologies. GPX-002 was developed by researchers at the University of Pittsburgh. Genprex’s diabetes gene therapy approach is comprised of a novel infusion process that uses an endoscope and an adeno-associated virus (AAV) vector to deliver Pdx1 and MafA genes to the pancreas. In models of Type 1 diabetes, the genes express proteins that transform alpha cells in the pancreas into functional beta-like cells, which can produce insulin but are distinct enough from beta cells to evade the body’s immune system. In Type 2 diabetes, where autoimmunity is not at play, it is believed that exhausted beta cells are also rejuvenated and replenished.

Tagrisso® is a registered trademark of AstraZeneca.

Keytruda® is a registered trademark of Merck & Co.