A Non-Lipid Nanoparticle Platform for Targeted RNA Therapeutic Delivery
Shur J, Kazuhiro I, Ombredane H, Sobolov S, Iyer K, Rapeport G.
Respiratory Drug Delivery 2025. Volume 1, 2025: 49-56.
Abstract:
The Nano-Emulsion Effective Delivery (NEED™) platform represents a promising alternative to lipid nanoparticles (LNPs) for delivering ribonucleic acid (RNA) therapeutics to the respiratory tract. This study demonstrates that NEED formulations maintain structural integrity during nebulization, exhibit improved tolerability, and ensure consistent RNA delivery across respiratory barriers. When used to deliver RIG-101, a synthetic RNA therapeutic based on stem-loop RNA design that acts as a potent retinoic acid-inducible gene I (RIG-I) agonist, NEED formulations showed superior immune activation and antiviral efficacy compared to conventional LNP formulations. Additionally, the NEED platform successfully delivered cystic fibrosis (CF) transmembrane conductance regulator mRNA (CFTR-mRNA) (RIG-301) to CF patient-derived airway organoids, resulting in significant functional improvement comparable to current standard-of-care therapy. The platform’s stability during aerosolization, coupled with enhanced transfection efficiency, highlights its potential as a transformative advancement in respiratory RNA therapeutic delivery for both infectious and genetic respiratory diseases.
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