Featured Publications

Prophylactic Vaccines
Title Journal Description
Multi-antigenic, pentamer-based, human cytomegalovirus mRNA vaccines that elicit potent humoral and cell-mediated immunity

Animal studies demonstrate that a challenging viral antigen, such as a multimeric, membrane-bound protein complex, encoded by mRNA, can elicit a robust, protective immune response.

A modified mRNA-based Ebola vaccine elicits a robust immune response and protects guinea pigs from a lethal challenge

This preclinical study with a LNP-formulated, modified mRNA vaccine demonstrates 100% survival and higher protective antibody titers than previously observed with viral vector vaccines.

Induction of robust B cell responses after Influenza mRNA vaccination is accompanied by circulating hemagglutinin-specific ICOS+PD-1+CXCR3+ T follicular helper cells

This non-human primate study of mRNA vaccines characterizes the cellular immune response that accompanies robust production of neutralizing antibodies, furthering our understanding of the immune signature associated with effective protection.

Rhesus macaque myeloid-derived suppressor cells demonstrate T cell inhibitory functions and are transiently increased after vaccination

Results in non-human primates characterize the response of immune-suppressive cells to vaccination, expanding our understanding of the mechanisms underlying vaccine immunogenicity versus reactogenicity.

Efficient targeting and activation of antigen-presenting cells in vivo after modified mRNA vaccine administration in rhesus macaques

This non-human primate study with mRNA vaccines characterizes the immune cells activated – and their subsequent priming of T cells – supporting our fundamental understanding of vaccine efficacy.

Vaccine Mediated Protection Against Zika Virus-Induced Congenital Disease

This research demonstrates that LNP-formulated mRNA vaccines can induce protection against viral placental and fetal damage in mouse models, critical for vaccines against viruses that exhibit vertical transmission.

Preclinical and clinical demonstration of immunogenicity by mRNA vaccines against H10N8 and H7N9 Influenza viruses

Two clinical studies demonstrate that an LNP-formulated, modified mRNA vaccine can elicit a protective immune response with an acceptable safety profile.

Modified mRNA vaccines protect against Zika virus infection

This in vivo research demonstrates that a mRNA vaccine encoding components of a subviral particle can prevent disease.

Overview of vaccine adjuvants: introduction, history, and current status (Review)

This review summarizes the current use and future prospects of adjuvants, which enhance the body's immune response to vaccines.

Cancer Vaccines
Title Journal Description
An RNA toolbox for cancer immunotherapy

This review highlights the potential for RNA cancer immunotherapies, including mRNA applications in cancer vaccines and immune modulation.

Local Regenerative Therapeutics
Title Journal Description
Biocompatible, Purified VEGF-A mRNA Improves Cardiac Function after Intracardiac Injection 1 Week Post-myocardial Infarction in Swine

Large animal studies demonstrate that direct injection of modified mRNA can produce local protein expression while maintaining immune silence, and can induce physiological effects at an organ level.

Modified mRNA directs the fate of heart progenitor cells and induces vascular regeneration after myocardial infarction

This foundational paper establishes that direct injection of modified mRNA in mice can induce local physiological changes and could be an effective approach for regenerative therapeutics.

Systemic Therapeutics
Title Journal Description
 Nature Medicine Messenger RNA as an etiological treatment for acute intermittent porphyria

Rapid onset of effect with mRNA therapeutics is demonstrated in the acute setting, imparting hepatic enzymatic activity with repeat dosing in small and large animals.

Systemic messenger RNA therapy as a treatment for methylmalonic acidemia

Results in a mouse disease model suggest that repeat systemic administration of LNP-formulated mRNA can elicit intracellular protein expression in hepatocytes for rapid and sustained amelioration of disease.

Platform
Title Journal Description
MicroRNAs Enable mRNA Therapeutics to Selectively Program Cancer Cells to Self-Destruct

Non-human primate and rodent studies demonstrate tissue-targeted mRNA translation and mitigation of potential off-target effects through the use of microRNA binding sites.

A Novel Amino Lipid Series for mRNA Delivery: Improved Endosomal Escape and Sustained Pharmacology and Safety in Non-human Primates

Non-human primate and rodent studies demonstrate the potential for repeat dosing of mRNA safely and at therapeutically relevant levels with proprietary Moderna lipids.

Lipid nanoparticle packaging is an effective and non-toxic mRNA delivery platform in embryonic zebrafish

This study suggests that direct injection of LNP-formulated mRNA may be a valuable tool in the study of developmental biology given transfection of distinct tissues in a developing vertebrate embryo.

Safety evaluation of lipid nanoparticle-formulated modified mRNA in the Sprague-Dawley rat and cynomolgus monkey

In vivo studies with systemic delivery of modified mRNA in an industry standard LNP demonstrate therapeutic levels of protein expression and predictable pharmacology and toxicology.

Boosting intracellular delivery of lipid nanoparticle-encapsulated mRNA

This research focuses on the mechanism of LNP endosomal escape, highlighting opportunities to increase LNP potency by improving the efficiency of escape.

Messenger RNA as a Novel Therapeutic Approach (Chapter)

This chapter highlights recent advances in the delivery of nucleotide therapeutics and their application to mRNA therapeutics.

A sensitivity analysis of RNA folding nearest neighbor parameters identifies a subset of free energy parameters with the greatest impact on RNA secondary structure prediction

This study explores the impact of folding energetics on mRNA secondary structure, contributing toward structure prediction and design of mRNA therapeutics.

N1-methyl-pseudouridine in mRNA enhances translation through eIF2α-dependent and independent mechanisms by increasing ribosome density

This paper explores the molecular mechanisms through which modified nucleosides can promote translation efficiency, which is critical to designing potent mRNA therapeutics.