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Targeted Therapeutics


Over the past decade, nanotechnology has emerged as a unique strategy for delivering novel drug candidates and for providing new formulations of matured therapeutics. Genisphere's 3DNA is a unique nanotechnology ideally suited for targeted drug delivery. The 3DNA nanostructure is biocompatible, easily adaptable, biodegradable and stable in circulation. Its DNA architecture enables the flexible and rapid attachment of therapeutic cargo and targeting molecules.

For a given disease, a therapeutic molecule and targeting molecule are selected and formulated with 3DNA. In the example pictured, 3DNA has been formulated with a small molecule and antibodies for targeting. This 3DNA formulation would then be administered systemically to the patient, via injection. The targeting molecule directs the 3DNA and its active drug molecule to the disease site. The 3DNA and therapeutic molecule are internalized by the cells at the site of disease. The targeted disease cell is either killed, or biologically modified, based on the therapeutic candidate administered using the 3DNA platform.


Targeting can be achieved by attaching specific antibodies, antibody fragments, peptides, sugars, or small molecules (vitamins, carbohydrates, lipids, bioactive molecules) to 3DNA. Since 3DNA is multivalent, two or more unique targeting devices may be used to deliver with the same nanocarrier. Pre-clinical data have demonstrated biodistribution profiles are determined by the chosen targeting moiety. Specific targeting molecules allow 3DNA transport across the blood-brain barrier and other physiological barriers to accumulate in targeted regions.


A variety of chemistries can be used to load therapeutic molecules onto the core 3DNA nanocarrier. Therapeutic antibodies and peptides are attached via an oligonucleotide linker that bridges the antibody or peptide to the 3DNA scaffold. Simple hybridization can be used to deliver siRNA, mRNA, and plasmid DNA using 3DNA. Small molecule therapeutics can be attached using standard linker chemistry or new strategies utilizing various chemistries available during nucleic acid synthesis or post synthesis modification. The multivalent properties of 3DNA also enable the attachment of multiple therapeutic molecules that may be the same or different. For example, 10s-100s of the same small drug may be attached to a single 3DNA or two different bioactive antibodies, peptides, or small molecules may be attached to modulate or amplify a therapeutic response. In oncology applications, 3DNA-based therapeutics have shown specific targeting of tumors, selective killing of targeted cells, and tumor shrinkage (result shown below generated in collaboration with Dr. Janet Sawicki at the Lankenau Institute for Medical Research).