Research includes innovations in lipid nanoparticle technology for mRNA delivery into hematopoietic stem and progenitor cells
Intracellular event-guided rational design of lipid nanoparticle
Assessment and prediction of SARS-CoV-2 variants for selection of COVID-19 booster vaccines
Moderna, Inc., (Nasdaq: MRNA) a biotechnology company pioneering messenger RNA (mRNA) therapeutics and vaccines, today announced new research being highlighted as part of the Company’s fourth annual Science Day. Moderna’s Science Day is designed to provide insight into the continued diverse efforts underway at Moderna to better understand how to use mRNA as medicines and vaccines and underscores the Company’s continued commitment to basic science and innovation.
“Science Day gives us an opportunity to provide insights into the advancements in our platform science and our further understanding of how to use mRNA as both a vaccine and a medicine. Our investments in basic science continue to result in major steps forward in our platform’s capabilities, and these have allowed us to open new therapeutic areas and new scientific directions,” said Stephen Hoge, M.D., President of Moderna. “Our investments in platform research along with our digital backbone and manufacturing plants have enabled us to create first-in-class mRNA medicines and vaccines. Today, we're excited to highlight our work to identify and address SARS-CoV-2 variants of concern, optimize our proprietary lipid nanoparticle (LNP) technology, and deliver mRNA to hematopoietic stem cells.”
Moderna currently has 24 mRNA development programs in its portfolio with 14 having entered clinical studies. The Company’s updated pipeline can be found at www.modernatx.com/pipeline. Moderna and collaborators have published more than 65 peer-reviewed papers.
At this year’s Science Day, Moderna will present new platform science and preclinical research, including:
mRNA Delivery to Hematopoietic Stem and Progenitor Cells (HSPC)
Of the nearly 30 trillion cells in the body, approximately 90% are of hematopoietic origin. Hematopoietic lineages originating in the bone marrow are intimately involved in maintaining homeostasis and human health. Nonetheless, there are hundreds of hematologic or immune-related disorders caused or exacerbated by cells of the hematopoietic lineage. In a disease setting, cells of hematopoietic origin interact with host tissues to drive chronic inflammatory and immune disorders. Because some hematopoietic stem and progenitor cells (HSPC) have self-renewal and pluripotent properties, targeting HSPC has the potential to modulate underlying chronic inflammation and immune-related disorders.
Advances in lipid nanoparticle (LNP) technology has allowed for delivery to the bone marrow following systemic LNP administration in vivo. This achievement has enabled Moderna scientists to deliver mRNA directly to bone marrow HSPC in vivo, leading to HSPC transfection and long-term modulation of all hematopoietic lineages. This represents a major milestone in impacting chronic inflammatory and immune related disease.
mRNA Engineering: Optimizing Ribosome Load
The ribosome plays a central role in orchestrating the life of mRNA inside the cell. Understanding how to modulate translation by changing mRNA sequence could enable a powerful lever to control the performance of an mRNA drug. To develop such a lever, the Moderna's scientists strove to characterize mechanistically how differences in translation drive differences in protein expression. In this research, an unexpected relationship emerged where mRNAs with low translation initiation rates conferred the highest and most durable levels of protein expression. By understanding the mechanisms that drive this behavior, the Company is taking mRNA design from a guess-and-check discipline into an engineering discipline. With tools in place, and more under development, Moderna is improving its ability to make an mRNA that generates the right amount of the right protein, for the right amount of time, in the right cell type. As Moderna builds these levers into its mRNA drugs, the Company believes it will be able to target more indications with more precision.
Addressing SARS-CoV-2 Variants of Concern
One part of the Company’s strategy to remain ahead of the SARS-CoV-2 virus is to closely monitor and address emerging variants of concern and waning immunity. Moderna is using artificial intelligence (AI) and machine learning to predict escape risk. This involves developing statistical models to understand and predict escape risk, including identifying breakthrough sequences from clinical trials and real-world data, examining spike protein biophysical and pseudovirus neutralization data, studying spike mutations and lineage info, and time tracking.
Science Day Webcast Information
Moderna will host its annual Science Day for analysts and investors at 8:00 a.m. ET on Thursday, May 27. A live webcast will be available under “Events and Presentations” in the Investors section of the Moderna website at investors.modernatx.com. A replay of the webcast will be archived on Moderna’s website for one year following the presentation.
In 10 years since its inception, Moderna has transformed from a science research-stage company advancing programs in the field of messenger RNA (mRNA), to an enterprise with a diverse clinical portfolio of vaccines and therapeutics across six modalities, a broad intellectual property portfolio in areas including mRNA and lipid nanoparticle formulation, and an integrated manufacturing plant that allows for both clinical and commercial production at scale and at unprecedented speed. Moderna maintains alliances with a broad range of domestic and overseas government and commercial collaborators, which has allowed for the pursuit of both groundbreaking science and rapid scaling of manufacturing. Most recently, Moderna’s capabilities have come together to allow the authorized use of one of the earliest and most-effective vaccines against the COVID-19 pandemic.
Moderna’s mRNA platform builds on continuous advances in basic and applied mRNA science, delivery technology and manufacturing, and has allowed the development of therapeutics and vaccines for infectious diseases, immuno-oncology, rare diseases, cardiovascular diseases and auto-immune diseases. Today, 24 development programs are underway across these therapeutic areas, with 14 programs having entered the clinic. Moderna has been named a top biopharmaceutical employer by Science for the past six years. To learn more, visit www.modernatx.com.
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including statements regarding: the potential for delivery of mRNA to hematopoietic stem and progenitor cells (“HSPC”) in vivo; methods of detecting and interrogating HSPC in vivo; the potential for delivering LNPs to hematopoietic stem cells in vivo; the ability to optimize codons and mRNA structures to increase total protein outputs; the potential for the Company to develop processes for controlling protein expression by modifying ribosomal loads; the Company’s ability to engineer LNPs capable of accessing difficult-to-transfect primary cells with efficient endosomal escape and high functional mRNA delivery; the Company’s strategy for combatting COVID-19, including processes for monitoring emerging variants and waning immunity; and strategies for modeling viral escape. 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; the fact that the rapid response technology in use by Moderna is still being developed and implemented; the safety, tolerability and efficacy profile of the Moderna COVID-19 Vaccine observed to date may change adversely in ongoing analyses of trial data or subsequent to commercialization; the Moderna COVID-19 Vaccine may prove less effective against variants of the SARS-CoV-2 virus, or the Company may be unsuccessful in developing future versions of its vaccine against these variants; despite having ongoing interactions with the FDA or other regulatory agencies, the FDA or such other regulatory agencies may not agree with the Company’s regulatory approval strategies, components of our filings, such as clinical trial designs, conduct and methodologies, or the sufficiency of data submitted; Moderna may encounter delays in meeting manufacturing or supply timelines or disruptions in its distribution plans for the Moderna COVID-19 Vaccine; whether and when any biologics license applications and/or additional emergency use authorization applications may be filed in various jurisdictions and ultimately approved by regulatory authorities; potential adverse impacts due to the global COVID-19 pandemic such as delays in regulatory review, manufacturing and clinical trials, 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 Annual Report on Form 10-K filed with the U.S. Securities and Exchange Commission (SEC) and in subsequent filings made by Moderna with the SEC, which are available on the SEC’s website at www.sec.gov. Except as required by law, Moderna disclaims any intention or responsibility for updating or revising any forward-looking statements contained in this press release in the event of new information, future developments or otherwise. These forward-looking statements are based on Moderna’s current expectations and speak only as of the date hereof.