On May 16, 2025, in New Orleans, LA, Dr. Bin Wu, Founder and CEO of Cytodigm, will present “Leveraging Novel PEG-free Lipid Nanoparticles for Tissue and Cell Targeting” at the 28th Annual Meetings of American Society of Cell & Gene Therapy.
Lipid nanoparticles (LNPs) are promising for delivering nucleic acids in gene and cell therapies, but conventional LNPs primarily accumulate in the liver, limiting targeting capabilities and causing toxicity. They also face challenges during nebulization for lung delivery and can cause immunogenic responses due to the presence of polyethylene glycol (PEG).
Cytodigm’s approach to address these challenges involves replacing PEG-lipid in LNPs with natural gangliosides, which can bind to Siglec receptors and enhance cell targeting. Results show that Cytofinity™ LNPs, while eliminating PEG-related issues, increase RNA delivery to bone marrow cells by 3.5 times, boost delivery to T cells by threefold, and reduce liver expression of payloads by ninefold. They maintain integrity during nebulization, with successful inhalation delivery demonstrated in mouse models. Immunohistochemistry confirmed successful transfection in various lung cells. Additionally, incorporating Cas9 and LoxP sgRNA enabled effective gene editing in bone marrow CD34+ cells.
In conclusion, Cytofinity™ LNPs provide a promising PEG-free alternative for delivering nucleic acids to extrahepatic tissues and cells, improving safety and efficacy in gene therapies.
