Science Overview

Our Science: Overview

Our targeted non-viral 3DNA® delivery platform is poised to transform the field of genetic medicines

The unique features of our proprietary 3DNA delivery platform enable targeted delivery of gene therapy, RNAi, and other genetic medicine modalities.

Multivalency

3DNA is a multivalent structure to which targeting molecules and nucleic acid based therapeutics can be attached. This property enables rapid optimization of both binding avidity and therapeutic potency and modularity, with high specificity of targeting. Ultimately, 3DNA expands options for delivering multiple modalities.

Unparalleled Tissue & Cell Targeting Specificity

3DNA leverages targeting molecules (such as peptides, antibodies, and small molecules) which bind to cell surface proteins expressed on the target cells, enabling high tissue specificity outside the liver with improved bioavailability. After binding to the target cell the 3DNA is internalized into the cell via a receptor-mediated process. By exploiting and withstanding the endocytotic process, the 3DNA delivers its genetic payload into the cytoplasm. When utilized to deliver gene therapy, the gene construct then localizes to the cell nucleus where a tissue specific promoter drives the expression of the desired protein. This unparalled tissue & cell targeting specificity eliminates the need for high doses and minimizes both dose related toxicity and off-target effects.

Delivery of Large Gene Constructs

3DNA is capable of efficient delivery of genetic medicines to cells, eliminating the size constraints imposed by a number of viral vector technologies. 3DNA has demonstrated the ability to deliver genes approaching 10kb, and we have yet to reach the upper limit for gene size that can be delivered. This means that we may be able to treat diseases that are currently untreatable.

Reduced Immunogenicity/Potential to Re-dose

3DNA has been designed to eliminate sequence specific elements from its scaffold that would otherwise trigger immune responses such as those initiated by toll-like receptors (TLR’s). Extensive preclinical data from repeat dosing studies in multiple species have shown the 3DNA scaffold itself does not generate neutralizing antibodies. This may allow for the ability to re-dose genetic medicines.

Rapid Formulation Development

Manufacturing of 3DNA is simple, reproducible, and scalable. 3DNA can be made in bulk, stored, and used in a modular process for formulating genetic medicines. Generating a final genetic medicine is a relatively straightforward process of taking the “off the shelf” 3DNA and attaching the relevant targeting molecules and the relevant nucleic acid based therapeutics.

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3DNA has shown efficacy in animal models targeting multiple cell types including brain microglia, lung endothelial cells, B cells, T cells, eye myofibroblasts, liver, muscle, pancreas, as well as various tumor cells. Discovery research is ongoing or planned in the ear, heart, and kidney.

In dose escalation and chronic dosing studies, 3DNA has demonstrated no toxicity either at the cell level or at the tissue/macro level. In preclinical safety studies, formulations combining 3DNA with both targeting molecules, as well as therapeutic cargo, have demonstrated strong safety profiles.

3DNA has demonstrated rapid and efficient biodistribution, in many cases accumulating in the cells of interest within minutes after intravenous injection.