What We Do

RNA Nanoparticle Delivery System (RNP)

Our RNA NanoParticle Delivery System (RNP) creates a unique nano-scaffold conjugated with small therapeutic RNA molecules and chemotherapeutic drugs that result in extraordinary pharmacokinetic and pharmacodynamic properties.

RNA NANOPARTICLE (RNP) PLATFORM:

• The negative charge of RNP prevents nonspecific binding to cells
• The defined structure and size (10-40nm) avoids immune system clearance
• Targets tumors through enhanced permeability and retention (EPR) effect and by active targeting of ligands on RNP
• Favorable pharmacokinetic profiles in mice:
  • T_1/2: 5-10hr compared to 0.25-0.76hr of siRNA
  • Biodistribution: mainly in the tumor but no other healthy organs.

• High delivery efficiency: ~5% compared to 0.7% using other nanoparticles
• Low toxicity: no toxicity after repeated IV up to 30mg/kg in mice
• Low immunogenicity: does not induce interferon or cytokine responses

RNA Nanoparticles Displaying Exosomes Delivery System (ExRNA)

ExRNA exosomes deliver nucleic acid-based therapeutics to treat life-threatening diseases. We harnessed the natural delivery capabilities of exosomes and specific targeting properties of RNA nanoparticles to create a novel class of biotherapeutics.

ExRNA PLATFORM:

• Displaying RNA nanoparticles on the exosome’s surface makes the ExRNA negatively charged, which prevents nonspecific binding to cells.
• Displaying RNA nanoparticles with an aptamer or chemical ligand enhances its targeting efficiency.
• ExRNA effectively delivers siRNA to the cell’s cytoplasm, escaping endosome trapping
• Validated in mice model systems