Moderna Announces Advancements in mRNA Platform Science for Application Across Multiple Diseases at Science and Technology Day - Seite 2

With Moderna's focus on respiratory diseases, the Company has invested in designing mRNA delivery systems tailored to targeting pulmonary disease. For pulmonary applications, delivery of mRNA medicines directly to the lungs by inhalation is the preferred method of administration in order to maximize the concentration of the medicine locally in the lung. Moderna researchers have developed a new LNP formulation that addresses many of the challenges associated with lung delivery of mRNA. This pulmonary LNP formulation is delivered by inhalation, uses multiple lipids, and is able to target specific areas of the lung. Moderna's optimization of pulmonary delivery of mRNA provides opportunities to address a range of unmet medical needs.

Moderna is collaborating with Vertex to deliver mRNA directly to the lungs via aerosolization to enable production of a functional cystic fibrosis (CF) transmembrane conductance regulator (CFTR) protein. CFTR is an important target for treatment because it is a defect in the CFTR gene that leads to buildup of thick mucus in the airways, chronic lung infections, inflammation and eventual respiratory failure. To date, the companies have completed IND studies that are supportive of advancing to clinical development. Vertex expects to submit an IND for this program in the second half of this year. In a second collaboration, the companies are collaborating to develop a portfolio of mRNA CF treatments, through the discovery and development of novel LNPs and mRNAs for the delivery of gene-editing therapies for the treatment of CF.

Characterizing Moderna's mRNA

mRNA is inherently unstable, but with continued investments in innovative science, Moderna researchers have developed a detailed understanding of the reactions that drive instability and lead to mRNA inactivation and degradation. mRNA stability has practical implications for product storage and shelf-life.

In addition to known chemical reactions that contribute to mRNA instability upon storage of product, Moderna researchers have identified and elucidated another mechanism that leads to a loss in mRNA activity: the formation of lipid-mRNA complexes, known as adducts. Since mRNA must be intact to be translated, adduct formation renders mRNA untranslatable, leading to a decrease in the expression of the proteins needed to treat or prevent disease.

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