Targeted-LNPs capable of functional DNA delivery to T cells enable in vivo CAR-T generation

Introduction:

We present a novel in vivo CAR-T approach using targeted lipid nanoparticles (tLNPs) co-loaded with transposon DNA encoding a CAR and SB100x transposase mRNA. Functionalized with T cell–specific binders, tLNPs enable efficient, specific delivery of both DNA and RNA to T cells. This non-viral platform offers a scalable, safer, and more accessible alternative to conventional CAR-T therapies requiring ex vivo cell manipulation which are expensive and logistically complex, limiting patient access (1).

Ator Ashoti – Senior Scientist, Nanocell Therapeutics; Jaime Bimbo – Scientist, Nanocell Therapeutics; Martijn Evers – Senior Scientist, Nanocell Therapeutics; Maurits Geerlings – CEO, Nanocell Therapeutics; Zhiyong Lei – Assistant Professor, UMC Utrecht; Jacek Lubelski – CTO, Nanocell Therapeutics; Suneel Narayanavari – Senior Scientist, Nanocell Therapeutics; Hanneke Rijssemus – Junior Scientist, Nanocell Therapeutics; Raymond Schiffelers – Professor, UMC Utrecht; Eline van Diest – Senior Scientist, Nanocell Therapeutics; Diana Vaz – Lab Manager, Nanocell Therapeutics; Christina Zotou – Junior Scientist, Nanocell Therapeutics