CybernaX presents a poster on conformation-adaptive lipids at the ASGCT 28th Annual Meeting
13 May 2025 — CybernaX presented a poster at the 28th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) in New Orleans, Louisiana, titled Conformation-adaptive Lipids for mRNA-Based Gene Editor Delivery via Lipid Nanoparticles (LNPs) in Non-Human Primates: Achieving a Wide Therapeutic Window. The abstract was accepted in the Nonviral Delivery category for lipid nanoparticles, and was presented by Dr. Pengbo Guo.
ASGCT 28th Annual Meeting, New Orleans.
Image: American Society of Gene & Cell Therapy.
13 May 2025 — CybernaX presented a poster at the 28th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) in New Orleans, Louisiana, titled Conformation-adaptive Lipids for mRNA-Based Gene Editor Delivery via Lipid Nanoparticles (LNPs) in Non-Human Primates: Achieving a Wide Therapeutic Window. The abstract was accepted in the Nonviral Delivery category for lipid nanoparticles, and was presented by Dr. Pengbo Guo.
Gene editing delivered intravenously needs a far higher dose than a vaccine does, which changes what the carrier has to do. Efficiency alone is not enough — the same particle has to be well tolerated and quiet immunologically at a dose several times higher than any approved LNP vaccine. That is the gap the poster addressed.
The work described a family of ionizable lipids designed so that their molecular conformation adapts to the pH change encountered inside the endosome, improving escape of the payload into the cytosol. Following library-scale screening and rodent studies, lead formulations were evaluated in non-human primates, where a single intravenous dose produced substantial editing of a liver target within a dose range that was well tolerated. Stability assessments over a six-month period supported the formulations’ handling and storage profile.
The poster was prepared with co-authors from CybernaX Biotechnologies, AccurEdit Therapeutics and Zhejiang University of Technology. Specific lipid identities, dose levels and efficiency figures are shared with partners under confidentiality.
About CybernaX
CybernaX Biotechnologies develops non-viral nucleic acid delivery technology, built on two lipid platforms: ELITE (CX Ionizable Lipid), an embodied-AI screened ionizable lipid library, and ENDURE (CX Stabilizing Lipid), a PEG-free monodisperse lipid built for long-term repeat dosing. The company licenses and co-develops its lipid platforms with biopharmaceutical and biotechnology partners worldwide.