New lipid nanoparticle shows muscle-specific mRNA delivery, reduces off-target effects
Source: Nano Werk, November 2023
(Nanowerk News) A team of researchers based at the University of Toronto’s (U of T) Leslie Dan Faculty of Pharmacy has discovered a novel ionizable lipid nanoparticle that enables muscle-focused mRNA delivery while minimizing off-target delivery to other tissues. The team also showed that mRNA delivered by the lipid nanoparticles investigated in their study triggered potent cellular-level immune responses as a proof-of-concept melanoma cancer vaccine.
Key Takeaways
Discovery of a novel ionizable lipid nanoparticle, iso-A11B5C1, enhancing muscle-focused mRNA delivery while reducing off-target effects in other tissues.
Iso-A11B5C1’s efficient mRNA delivery to muscle tissues triggers potent cellular immune responses, showcasing potential as a melanoma cancer vaccine.
The study challenges existing beliefs, demonstrating effective stimulation of cellular immune response and anti-tumor effects without lymph node targeting.
Comparison with existing mRNA delivery systems reveals iso-A11B5C1’s superior muscle-specific efficiency and different immune response, valuable for cancer vaccine development.
Introduction of a rapid, precise platform for creating diverse ionizable lipids, accelerating the development of RNA-based therapies.
The Research
The study, led by Bowen Li, assistant professor, Leslie Dan Faculty of Pharmacy, U of T, was published in Proceedings of the National Academy of Sciences (“Combinatorial design of ionizable lipid nanoparticles for muscle-selective mRNA delivery with minimized off-target effects").