Moderna Highlights Advances in Platform Science and Innovative Vaccine Research at Third Annual Science Day
Research includes innovations in mRNA and protein engineering and delivery science to improve therapeutic properties
Highlights research results from ongoing collaborations with
“Science Day is an opportunity for us to provide insights into the advancements in our platform science and our further understanding of how to use mRNA as a medicine. Our substantial investments in basic science to date have resulted in major steps forward in our platform’s capabilities, and these have allowed us to open new therapeutic areas and new scientific directions,” said
At this year’s Science Day,
Engineering mRNA and Proteins to
An advantage of mRNA medicines over small molecule drugs is the ease of engineering their properties to achieve desired pharmacology. New research will be presented on efforts to engineer both mRNAs and encoded proteins to improve and extend therapeutic effect.
These efforts include modifications to mRNA and proteins to prevent degradation and lengthen half-life. Presentations will include data on the addition of an inverted deoxythymidine (idT) to the 3’-end of mRNA, which is designed to stabilize mRNA by blocking its degradation through deadenylation. Preclinical data show that 3’-idT-stabilized mRNA encoding phenylalanine hydroxylase (PAH), the enzyme missing or dysfunctional in the metabolic disorder phenylketonuria (PKU), supports sustained reduction of serum phenylalanine levels in a PKU mouse model. Data will also be presented on removal of ubiquitination sites from PAH, designed to prevent ubiquitin-mediated protein degradation. Results show that combining mRNA stabilization along with PAH stabilization maintained low serum phenylalanine levels even further, suggesting an additive effect.
One challenge for the production of mRNA therapeutics is to minimize or eliminate double-stranded RNA (dsRNA) impurities produced during the synthesis process, as this can trigger undesirable innate immune responses in vivo. At Science Day,
Optimizing Lipid Nanoparticle Technology
The Company will present new research in delivery science to optimize its lipid nanoparticles (LNPs). The presentations will highlight a novel squaramide-based ionizable lipid designed to enhance the interactions between the lipid and mRNA. Preclinical data from new LNPs incorporating this novel ionizable lipid show improved protein expression after IV administration, including repeat dosing, and efficient delivery to the liver, as compared to current state-of-the-art
This squaramide-based LNP represents a new delivery system and will be used with the Company’s glycogen storage disease type 1a (GSD1a) candidate (mRNA-3745), which is in preclinical development. Preclinical studies with this new LNP containing mRNA encoding for G6Pase, the missing or dysfunctional enzyme in GSD1a, show sustained improvements in fasting blood glucose in a mouse model of GSD1a, achieving the target product profile.
Collaborating on an HIV Vaccine
Science Day will also include presentations from
Science Day Webcast Information
Forward Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including regarding the Company’s mRNA and encoded protein engineering efforts, the Company’s new squaramide-based LNP and its expected use with mRNA-3745 and other new development candidates, the potential for the Company’s mRNA platform to significantly accelerate early clinical trials and transform HIV vaccine science and development efforts, and the presentation of research and data at the Company’s upcoming Science Day. In some cases, forward-looking statements can be identified by terminology such as “will,” “may,” “should,” “could”, “expects,” “intends,” “plans,” “aims,” “anticipates,” “believes,” “estimates,” “predicts,” “potential,” “continue,” or the negative of these terms or other comparable terminology, although not all forward-looking statements contain these words. The forward-looking statements in this press release are neither promises nor guarantees, and you should not place undue reliance on these forward-looking statements because they involve known and unknown risks, uncertainties, and other factors, many of which are beyond Moderna’s control and which could cause actual results to differ materially from those expressed or implied by these forward-looking statements. These risks, uncertainties, and other factors include, among others: the fact that there has never been a commercial product utilizing mRNA technology approved for use; potential adverse impacts due to the global COVID-19 pandemic such as delays in regulatory review, manufacturing and supply chain interruptions, adverse effects on healthcare systems and disruption of the global economy; and those other risks and uncertainties described under the heading “Risk Factors” in Moderna’s most recent Quarterly Report on Form 10-Q filed with the
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