INV-16006
Oligonucleotide-based gene therapy holds tremendous promise for treating a variety of disorders with a genetic basis, including cancers, neurological diseases, and metabolic conditions. However, due to difficulties including poor accumulation at target sites, removal by immune responses, nuclease degradation, and low biochemical efficacy few therapies are available commercially. To overcome these shortcomings, complexation of nucleic acids with cationic and noncationic polymers have been used but the problems with the immunogenic response and low residence time in blood persist. Hence, there is a need for a robust and efficient delivery system for the use of oligonucleotides as therapeutics and diagnostic agents.
The present technology is a novel drug delivery system in the form of a brush polymer-DNA conjugate, with high selectivity and stability. The oligonucleotides (DNA) are covalently linked to the backbone of a non-cationic, biocompatible brush polymer (polyethylene glycol (PEG), polysaccharide or zwitterion polymer). The compaction of DNA by high-density side chains of brush polymers provide stability and selectivity to the DNA by steric hindrance.