New partnerships could help bring a novel class of biopharmaceutical to patients. Messenger RNAsmolecules that carry information between the genome and the protein-building machinery of cellscould become the next big class of biopharmaceutical drug. These molecules were once thought unusable as drugs because they are notoriously fragile and could rouse an inflammatory immune response. Now, thanks to several recent scientific discoveries, they are attracting the increasing attention of pharmaceutical companies. Recently, the pharmaceutical heavyweight AstraZeneca announced it would pay Moderna Therapetuics $240 million for rights to the startups mRNA technology, which it will use to develop treatments for cardiovascular, metabolic, and cancer patients. Inside a cell, mRNA serves as an intermediary between DNA-encoding genes and their protein products. As a drug, an mRNA would supply the biological instructions for producing a protein inside cells, perhaps a protein that replaces a missing or broken version inherited as part of a genetic disorder. In some ways, an mRNA would be more efficient than DNA-based gene therapy (which would require the cells to make their own mRNA intermediary before producing a protein) and more effective than recombinant protein therapy. While several companies are developing therapeutic gene-silencing RNAs, which inhibit damaging proteins from being … Continue reading
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