Innovative technology platform
The NanoDe delivery technology is a novel non-viral, non-lipid nucleic acid delivery system, that:
- Encapsulates/ delivers oligos, RNA, DNA
- Infiltrates difficult-to-deliver tissues including the CNS (crosses BBB)
- Delivers with great efficiency, thus lowers dosing and improving safety
- Targets cells and tissues via cellular receptor
- Has demonstrated efficacy in multiple animal disease models
- Is expected to be cost effective in scale-up
Overcomes challenges such as
- Sequestration by liver
- Biological barriers
- Degradation by nucleases
- Endosomal trapping
Structure of assembled Nanopieces
- Formed by self-assembly of proprietary small molecule nanomaterial & nucleic acid.
- Non-covalent ‘sandwich’ of nucleic acids between a nanotube ‘core’ and shell.
- Cytoplasmic pH-triggered disassembly is believed to cause spontaneous nucleic acid release inside cells.
- Bench-scale process for production has been developed and steps are amenable to scale up.
Nanopiece (side view)
Nanopiece (top view)