Novel compositions of nanoparticles for nucleic acid therapeutic delivery
T2018-222 Novel lipid-based nanoparticles that utilize a surfactant to improve self-assembly and stability. Finalized particles can be used for delivery of nucleic acids such as oligonucleotides and plasmid DNA.
Lipid-based nanoparticles (LNPs) have been extensively utilized in nucleic acid delivery. However, there are a number of challenges associated with LNP formulation. It is difficult to make sub-100-nm LNPs with a high nucleic acid concentration by traditional self-assembly methods. LNP synthesis typically requires 40% ethanol during assembly, which needs to be removed afterwards. These requirements complicate the manufacturing of LNP and reduce use for large-scale applications and clinical translation of LNP formulations of nucleic acid therapeutics.
Dr. Robert Lee at The Ohio State University has developed a novel formulation for creation of lipid-based nanoparticles using surfactants which eliminates a need for ethanol. In addition, the nanoparticles developed are controllable in size and can deliver a high concentration of nucleic acids. Proof-of-concept studies in the laboratory utilized oligonucleotides (ON) as the nucleic acid and found that high concentrations of ON were delivered using the particles with a low cytotoxicity and hemolytic activity.
- For delivery of nucleic acids such as siRNA, antisense oligos, miRNA, mRNA
- Large-scale production of nucleic acid loaded nanoparticles for therapeutic treatments
- Synthesized in ethanol-free protocol
- Enhanced colloidal stability
The Ohio State University laboratory that developed this technology has expertise in a range of areas related to lipid nanoparticles. They specialize in custom-design LNP for various cargo and in developing products tailored to the specific clinical application. The lab is focused on nucleic acid drug delivery and can be used for mRNA, plasmid DNA, siRNA, miRNA, antisense ODN, CpG ODNs and sgRNA for CRISPR gene editing, and any gene therapy related applications. The laboratory is open for collaboration for additional cargos and investigational routes.