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Pipeline

Overview

The flexibility of the 3DNA platform enables application in gene delivery, biologics, small molecules, and RNAi therapeutics. Through collaboration and on its own accord, Genisphere has advanced several 3DNA-based lead compounds. 3DNA Pipeline

Oncology

GL-233

Ovarian cancer is the most deadly gynecologic cancer. Human antigen R (HuR, also known as ELAVL1) is a post-transcriptional regulatory RNA-binding protein that regulates the expression of multiple genes known to function in tumor cell survival and in drug resistance. It is abundant and hyper-functional in ovarian cancer cells as compared to normal cells, thus providing a therapeutic window. A unique siRNA is used in GL-233 to suppress HuR. Ovarian tumor-bearing mice treated with GL-233 showed extended survival and no overt toxicity. GL-233 is being investigated as a complementary approach to current, active therapies for ovarian cancer for overcoming drug resistance and inhibiting tumor growth

Delivery of Therapeutics Targeting the mRNA-Binding Protein HuR Using 3DNA Nanocarriers Suppresses Ovarian Tumor Growth
Cancer Research 2016;76(6);1-11
doi: 10.1158/0008-5472.CAN-15-2073

Ocular

GL-249

Cataract surgery is one of the most common procedures performed throughout the world, but it is not without complications. A condition called Posterior Capsule Opacification (PCO) is a vision impairing disease that occurs in some adults and in most children following cataract surgery. PCO arises when myofibroblast cells grow in the capsule surrounding the surgical site, and form cloudy scar tissue inside the eye that blocks vision. While PCO is treated readily by laser therapy, the procedure is neither risk free nor available in the developing world, regions with the highest incidence of blindness due to cataracts. GL-249 is a preventative treatment for PCO, designed to be administered at the time of cataract surgery. The unique antibody used in GL-249 directs 3DNA to deliver the cytotoxin doxorubicin only to the problematic myofibroblast cells. Rabbits that underwent cataract surgery and were treated with GL-249 at the time of surgery showed reduced PCO compared to rabbits that did not see the drug.

chart: Central and Peripheral PCO Scores for Various GL-249 Treatments and Control

Antibody Conjugated, DNA-Based Nanocarriers Intercalated with Doxorubicin Eliminate Myofibroblasts in Explants of Human Lens Tissue
The Journal of Pharmacology and Experimental Therapeutics 2017;jpet.116.239079
doi: 10.1124/jpet.116.239079

PDF icon Reducing Posterior Capsule Opacification by Eliminating Myo/Nog Cells Using 3DNA Nanocarriers and the G8 Antibody Association for Research in Vision and Opthamology (ARVO) Poster, May 2015

PDF icon Long Term Culture of Human Lens Tissue for Screening Drugs with the Potential to Prevent PCO Association for Research in Vision and Opthamology (ARVO) Poster, May 2016