Nanoparticle Composition for Nucleic Acid Delivery
T2013-246 Lipid nanoparticles (LNs) usable for the delivery of therapeutic compositions, including, but not limited to nucleic acids (NAs).
Nucleic Acid (NA) based therapies are used to inhibit gene expression. Lipid nanoparticles (LNs) are capable of carrying these therapies into cells. However, this delivery requires overcoming several physiological barriers. For instance, one barrier is the acidic endosome conditions within the cell. LNs must release the drug into the cytoplasm before the conditions degrade the drug. In addition, NAs have high negative charges that hinder their passage across the cell membrane. Therefore, there is a need for efficient delivery methods that protect and transport the therapies effectively. New LNs are further needed to transverse the capillary endothelium, reach target cells without causing excessive immunoactivation or off target cytotoxicity, promote endocytosis, and form stable formulations with long shelf-lives in order to promote this efficiency.
Ohio State Inventors, led by Dr. Robert J. Lee, developed a LN compositions that overcome the barriers that traditional therapeutic and NA-based therapies face. The LNs comprise a mixture of lipids, along with PEGylating lipid and/or cholesterol, which improves transfection activity and reduces cytotoxicity. The LNs are also stable under refrigeration for several months. The compositions also include positively charged quaternary or tertiary amines-based cationic lipids to combat endosomal escape of the therapies.
- Pharmaceutical Drug Therapy
- Pathology Research
- Medical Laboratories
- Efficient delivery of therapies
- Long shelf life
- Stable formulations
Patents have been granted/pending: Australia, Brazil, Canada, China, EPO, India, Japan, Republic of Korea, Mexico, USA
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.