TECHNOLOGY

Bio-Path’s neutral lipid delivery technology provides systemic distribution of nucleic acid drugs throughout the human body through simple intravenous transfusion. Bio-Path’s delivery technology applies to both double stranded (siRNA) and single stranded (antisense) nucleic acid drugs. This technology has the potential to revolutionize the treatment of cancer and other diseases where the targets of disease are well characterized. The reason is because the neutral lipid delivery capability enables systemic delivery of nucleic acid drugs to diseased cells, something no other technology can do.

Delivery of siRNA Molecules: Small interfering RNAs, or siRNAs, are the molecules that mediate RNA interference (“RNAi”) and interfere with the process of producing proteins inside cells. The siRNA are short double stranded nucleic acid molecules that can be synthesized chemically and introduced into cells, creating enormous possibilities for using siRNAs as drug candidates to treat major diseases including cancer. Introduction of a double stranded nucleic acid molecule specific to a target disease-causing protein conditions cellular machinery to shut-down production of that target protein. Potential advantages of siRNA drugs include:

The ability to target virtually any disease-causing protein where the targets of the disease are well-characterized.
With siRNA therapeutics, it is possible to block the production of disease-causing proteins, which provides improved capability for disease control.
Because siRNA are designed to target a specific disease-causing protein, the potential for other interactions that might cause toxicity is minimized.

However, the greatest challenge facing successful deployment of siRNA drugs has been lack of technology that can deliver the double stranded nucleic acid siRNA molecules to the target diseased cells. Bio-Path’s patented delivery technology solves this problem by employing DOPC neutral lipid formulations to incorporate the siRNA into liposomes. These liposomes are inert in the body, incorporate the siRNA with high efficiency and form nano-sized particles that can migrate inside tumors.

Testing of Bio-Path’s delivery technology in animals has demonstrated a 10-30 fold advantage in delivering siRNA to target cancer cells.

Delivery of Single Strand Nucleic Acids: Similar to siRNA, a second class of RNAi drugs involving single strand nucleic acid molecules has been developed by the pharmaceutical industry. Introduction of a single strand nucleic acid molecule specific to a target disease-causing protein blocks the ability of the cellular machinery to produce that target protein. However, delivery of single strand nucleic acid molecules to target cells has been a challenge that has limited pharmaceutical industry development of effective drug products employing this technology. Bio-Path has patented delivery technology that solves this problem by employing a DOPC neutral lipid formulation for single strand molecules that incorporate into liposomes.