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MRNA / LNP / RESEARCH LIBRARY
Important mRNA/LNP publications from the past 120 days
Search bilingual publication guides on delivery, in vivo CAR-T, therapeutic translation, safety, analytics, and manufacturing.
Nine bile-acid-derived sterols varied hydroxylation and alkyl-tail chemistry to probe LNP organization and organ expression. Molecular simulations and experiments linked membrane descriptors with encapsulation and formulation behavior. Replacing cholesterol shifted expression toward the spleen, and a lead sterol enhanced antigen-specific responses. The work offers a preclinical formulation framework rather than validated prediction of human biodistribution.
Immunomic announced first-patient dosing in VITALITI, the first-in-human Phase 1 trial of ITI-5000, a self-amplifying RNA cancer vaccine. The open-label study evaluates safety, tolerability, and preliminary immune activity in stage II–III triple-negative breast cancer, alone or alongside standard adjuvant therapy. Initial monitoring of the first participant identified no significant safety concerns, but this milestone provides no efficacy evidence.
Bispecific antibodies were used to bridge PEG motifs on LNPs and selected cellular surface markers without elaborate chemical conjugation. The platform enabled targeted mRNA delivery in cell models and transferrin-receptor-associated delivery in vivo. Researchers also measured endosomal responses at different antibody-loading ratios. Splenic enrichment supports further immune-cell targeting studies, but does not establish a clinical delivery solution.
A hollow-fiber membrane platform uses dense nanopore arrays to control localized mixing during lipid nanoparticle assembly. The continuous process generated tunable particle sizes, narrow distributions, and high encapsulation, with particle properties linked to membrane pore size. Multiple lipid chemistries and nucleic acid payloads were compatible, and cell and animal studies supported activity. Commercial GMP performance remains to be established.
Acid-cleavable cholesterol-derived crosslinkers and PEG-diamines stabilized anionic LNPs that otherwise lost activity during purification. By tuning crosslinking architecture, researchers balanced structural integrity with RNA release and improved splenic mRNA expression. Mechanistic analyses implicated intracellular processing as well as release behavior. These formulation-specific animal results do not establish general T-cell targeting or clinical manufacturing readiness.
Tempest outlined TPST-4003, an in vivo CAR-T candidate using CD7-targeted mRNA/LNP delivery of a dual CD19/BCMA CAR. A planned investigator-initiated study would enroll approximately ten patients with nervous-system autoimmune diseases, initially including myasthenia gravis and multiple sclerosis, with dosing anticipated in the fourth quarter. This is a development plan, not human efficacy evidence for the in vivo candidate.
A library of 56 ionizable lipids combined biodegradable disulfide linkages with different hydrophobic tail lengths and branching patterns. Structure-activity testing linked tail architecture to endosomal escape, translation, and biocompatibility. Optimized particles reduced cytotoxicity and cytokine induction, with immune-response evaluation extending to a nonhuman primate model. These findings support lipid design, rather than establishing human safety.
Starting with a liver-tropic lead from 90 degradable lipidoids, researchers attached small-molecule ligands to redirect delivery toward the lung. The modified particles showed increased vitronectin association and enhanced pulmonary mRNA expression, with genome-editing proof of concept. This modular chemical approach retains the parent scaffold, but its targeting performance remains demonstrated in preclinical experimental conditions.
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