Tunable, non-lipid-based compositions for carrier-free delivery of therapeutic biopolymers such as RNA.
Membrane-less organelles (MLOs), which are fluid compartments formed from proteins and RNA through liquid-liquid phase separation, serve important biological functions within cells. In an effort to better understand the forces driving their formation and alteration, a variety of hollow, lipid-free, vesicle-like compositions and methods for their preparation have been developed.
This invention provides polyionic vesicles comprising a layer made up of oppositely charged (bio)polymers. These lipid-free vesicles are referred to as membrane-like nucleoprotein assemblies (MNAs). MNAs exhibit structural and functional similarities to liposomes, including an ability to selectively encapsulate biomolecules. More generally, these highly tunable vesicles, which comprise a rim layer that defines an interior volume, exhibit molecular ordering, size-dependent permeability, selective encapsulation and complex surface topology. These MNAs may be used for drug delivery and/or as tunable, stimuli-responsive release/trapping systems. Drug delivery applications can employ carrier-free vesicles, wherein a therapeutic biopolymer (e.g. RNA, DNA, protein, etc.) is provided as a component of the rim structure, and/or drug-loaded vesicles, wherein therapeutic cargo is present within the lumen. Beyond therapeutics, agricultural biotechnology applications, including the release of insecticides and/or pesticides, are also possible through the use of these novel vesicles.
Publication Link: bioRxiv 2020
These MNAs can be prepared without the use of organic solvents, unlike liposomes. In drug-delivery applications, these compositions enable carrier-free delivery of therapeutic biopolymers, including but not limited to RNA.
• Drug delivery
• Agricultural biotechnology
Intellectual Property Summary:
US Provisional Patent Application 62/958,039 filed January 7, 2020.
Stage of Development: In vitro demonstration.
Licensing Status: Available for licensing or collaboration.