Sequence to Substance: Making the mRNA Therapeutic

mRNA-based therapeutics are being explored across a range of applications, including vaccines, protein replacement and immunotherapies (2).

Before any formulation decisions enter the picture, teams need confidence in the RNA itself: that it is the right sequence, right properties and the right purity to behave predictably downstream. That is where it helps to separate drug substance from drug product. The drug substance is the active ingredient intended to deliver a pharmacological effect, while drug product is the finished dosage form that contains that ingredient (6).

This post focuses on what happens upstream, making the mRNA drug substance before formulation. In practical terms, that upstream work spans choosing an mRNA construct, producing it by IVT, and then purifying and analyzing the product so it has the desired quality attributes (5).

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Total Eclipse of the CAR T: How mRNA Vaccine Technology Can Be Used to Help Heal “Broken” Hearts

While you can rely on Taylor Swift and Adele to help heal emotional heartbreak, unfortunately treating a physically “broken” heart, a heart damaged by fibrosis, is a much more complicated process than putting on your favorite sad songs and wallowing in your feelings. In a recent study published in Science, researchers developed a therapeutic approach to treat damaged hearts in mice through the removal of scar tissue using genetically engineered immune cells (CAR T cells) and the mRNA technology used in the mRNA coronavirus vaccines.

Genetically engineered CAR T cells have been used l for repairing damaged hearts in mice
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