Publications

This pre-clinical study shows that an mRNA vaccine protects against viral infection in transgenic mice modeling human antigen presentation to T cells.

Phase 1 clinical studies of the first mRNA vaccines against H10N8 and H7N9 influenza viruses elicited robust immune responses, further supporting the potential of mRNA as a vaccine platform.

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.

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.

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.

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

Data from a Phase 1a/b study of mRNA encoding for vascular endothelial growth factor A (VEGF-A) shows tolerability, protein expression and protein pharmacology in human subjects and its potential for use as a regenerative therapeutic.

In mouse models, the intradermal administration of mRNA encoding for VEGF-A can induce a localized, sustained, and reproducible biological response, including blood vessel formation and oxygenation to accelerate diabetic wound healing.

(This publication is the work of a Moderna strategic collaborator and Moderna assumes no responsibility for the information or statements therein).

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.

This pre-clinical study shows mRNA encoding the alpha 1-antitrypsin (AAT) protein led to functional protein expression in human AAT deficient patient hepatocytes and mouse models of AAT deficiency, supporting the translation of mRNA in hepatocytes and secretion of AAT protein into systemic circulation as a potential replacement therapy.

This publication outlines a translational modeling framework that can be used to describe the effects of mRNA therapy for acute intermittent porphyria (AIP) in preclinical models and support development in humans.

This pre-clinical study shows that repeat dosing of mRNA encoding ARG1 delivered via lipid nanoparticles was able to treat mouse models of arginase deficiency and supports the potential of mRNA therapeutics to treat rare metabolic disorders.

Creation of a genetic mouse model that resembles much of the clinical phenotype of rare disease patients demonstrates the value of translational research in developing novel therapies for rare diseases.

A systematic review of the global epidemiology literature on MMA confirms that MMA and its subtypes are ultra-rare with similar detection rates across geographic regions with the exception of the Middle East, where the disease is more frequent.

A systematic review of the global epidemiology literature on PA confirms that PA is an ultra-rare disorder with similar detection rates across geographic regions with the exception of the Middle East, where the disease is more frequent.

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.

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

This study shows that the structure of the mRNA coding sequence regulates protein expression through changes in functional mRNA half-life, highlighting an important role of mRNA secondary structure in regulating mRNA stability and potentially enabling enhanced control of protein expression levels for mRNA therapeutics.

This study describes a deep learning model of 5’ UTR impact on protein expression, demonstrating the ability to optimize expression levels with mRNA therapeutics.

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.

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.

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

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

Local delivery of mRNA encoding the secreted cytokines IL-23 and IL-36γ and the membrane-bound T-cell co-stimulator OX40L induced a broad immune response promoting tumor regression in both injected lesions and distant un-injected tumors in mice.