Key Words:
lipid nanoparticle, kidney, gene therapy, siRNA, mesoscale nanoparticles
Polymeric Mesoscale Nanoparticles (MNPs) are the subject of CUNY CTO reference 24A0012. That IP focuses on the use of 300-500 nm polymeric nanoparticles to deliver therapeutics directly to the kidney. The MNPs localize to the continues due to their size and surface PEGylation, but not due to the nanoparticle composition. That application focuses on the use of a PLGA-PEG polymeric composition of the particles, but does not protect other particle compositions. Therefore, it is necessary to protect other forms of particle composition that would commonly be used to deliver therapeutics. The most common nanoparticle composition found in the literature and on the market is a lipid nanoparticle. Further, lipid nanoparticles are widely used as they often exhibit higher nucleic acid loading potential and quicker drug release compared to polymeric particle compositions. Thus, we sought to create lipid nanoparticles that are mesoscale in range and use them to deliver small molecules and functional nucleic acids to the kidneys.
Mesoscale lipid nanoparticles (MLNPs) can be used to deliver nucleic acid therapeutics selectively to the kidneys, which is potentially useful as a therapeutic strategy for kidney disease treatment. MLNPs are a drug delivery system that accumulates in the kidneys several fold more than in other organs due to their size (in the range of 300 – 500 nm) and surface chemistry. This presents a considerable advantage compared to conventional therapies for kidney diseases since drug uptake in the kidneys is generally low due to their physiological excretory function. Selective drug delivery can help achieve higher drug concentrations in the kidneys, thus improving the efficacy of treatment. In addition, targeted delivery to the disease site reduces the adverse effects of the drug in healthy tissues, allowing for higher doses of the drug to be administered. The localization of mesoscale nanoparticles (MNPs) within the kidney was previously shown to be in the proximal tubules1,2, therefore, this system would potentially be applicable to the treatment of any etiology of kidney disease primarily affecting the tubules, though altered glomerular filtration barrier-associated diseases represent an additional route for glomerular targeting.
MLNPs are specifically designed to encapsulate nucleic acids. Nucleic acid therapeutics are a novel treatment modality that can expand the capabilities of current treatments. However, delivery of nucleic acids to the disease site presents a major challenge due to their susceptibility to enzymatic degradation, immunogenicity, and inability to cross cell membranes3. MLNP drug delivery system provides greater stability of the nucleic acid cargo in the biological environment, ensuring its intact delivery to the disease site, as well as enabling its transport through the cell membrane. Our initial demonstration focuses on the delivery of small interfering RNA (siRNA), but MLNPs can potentially be used for the delivery of any modality of nucleic acid therapeutics, including messenger RNA (mRNA), antisense oligonucleotides (ASO), plasmid DNA (pDNA), etc. Thus, this system can be generalized for numerous drug delivery applications in the treatment of kidney diseases.
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