-Negative charge disallows nonspecific permeation of negatively charged cell membrane
-Controlled synthesis provides defined structure and stoichiometry
-Multi-valency allows combination therapy and simultaneous targeting and detection
-Targeted delivery allows receptor mediated endocytosis
-Advantageous size (10 – 40 nm)
-Extended in vivo half-life (5-12 hr compared to 15-45 min for siRNA)
-Avoidance of antibody induction (protein-free) allows repeated treatment for chronic diseases
- Favorable pharmacokinetic profile in mice:
Half-life (T1/2) : 5-10 hr compared to 0.25-0.76 hr of siRNA counterpart;
Clearance (Cl): <0.13 l/kg/hr,
Volume of distribution (V(d)): 1.2 l/kg;
Does not induce interferon (IFN) response or cytokine production；
Repeated IV administrations of up to 30 mg/kg do not result in toxicity.
- RNA nanoparticles are classified as chemical drugs rather than biologics. This classification facilitates drug approval.