Small interfering RNA (siRNA) therapies
Small interfering RNA, or siRNA, are naturally occurring molecules that act within the RNA interference pathway to regulate protein production and can be designed to block translation of disease-causing proteins. siRNA therapies are designed to be highly selective, working precisely at the desired target and minimizing the risk of off-target effects
The lack of efficacious and universal tools for Delivery of genetic drugs is widely recognized as the major factor holding back their clinical implementation. The problem is that genetic drugs such as RNAi are relatively large and carry negative charges, preventing their transport across the cell membrane to the target site within the cell.
The Membrane Dipole Potential
The membrane dipole potential is a recently discovered voltage, measurable in every leaflet of any phospholipid membrane. Due to the high hydrophobicity of biological membranes, the dipole potential translates into an enormously strong intra membrane electric field of up to 1 billion V/m (!), spanning ~30 Å, from the membrane surface to the membrane center.
Aposense’s Molecular Nano-Motors
Fascinated by the fact that every cell harbors its own discrete and strong powerhouse, Aposense successfully developed novel Molecular Nano-Motors, being novel small-molecule chemical entities, capable of “energy mining” from the Membrane’s Dipole Potential, and its translation into kinetic energy, for movement within the hydrophobic membrane core. This innovative Molecular Nano-Motors are the core of our technology.
Upon linkage to a cargo drug, this intra membrane movement of the MNMs is utilized for the trans-membrane delivery of membrane delivery of siRNA into the cytoplasm.
Within the cytoplasm, a unique and specific detachment mechanism cleaves the MNMs leaving siRNA to exert its gene-silencing function, preventing the over production of disease-causing proteins by siRNA.
Creating a new industry standard for the delivery of genetic drugs. The MNMs are modular, can be “click attached” to any oligonucleotide sequence while its unique distribution profile may unlock novel targets for genetic therapeutics.