Background

Nucleic acid therapeutics promise to treat genetic disorders, infectious disease and cancer. However, nucleic acid delivery to target cells remains a challenge, as nucleic acids are highly charged and easily degraded. Viral vectors have been used to accomplish this task but have many drawbacks including the efficiency of entering the cells and possible side effects of the vector itself. There has been intense work to develop better delivery systems to make good on the promise of nucleic acid therapeutics.

A nucleic acid delivery vehicle must be able to: provide stable encapsulation, proceed easily through the blood, and target a specific tissue. Target cells must readily take up the delivery vehicle to ensure that the genetic material is appropriately delivered. And finally, the delivery vehicle should be non-toxic and able to be broken down and excreted easily after material delivery.

Polymeric Nanoparticles (PNPs) or micelles, have become a focus as a nucleic acid delivery tool as they are relatively stable, functionalizable and tunable.

Technology Overview

Here, researchers have developed a polymeric nanoparticle with a hydrophobic core using a two step process to create a hierarchical assembly of block copolymers. Step one, the nucleic acid cargo electrostatically binds to a block co-polymer of polycaprolactone and polyvinyl pyridine forming a polyplex with surrounding polycaprolactone chains. Step two, the polyplex is microprecipitated with a copolymer of polycaprolactone and poly ethylene glycol (PEG). Step two causes the polycaprolactone chains to condense around the genetic material creating a very stable environment that is surrounded by PEG creating a hydrophilic shell.

Benefits

• stable encapsulation
• potential for use in gene therapy or vaccines
• employs microfluidics for particle creation

Applications

• gene therapy
• vaccine delivery
• drug delivery

Opportunity

Provisional patent application filed.

Seeking a development partner of licensing partner.