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Generation Bio is innovating genetic medicines to provide durable, redosable treatments for people living with rare and prevalent diseases. The company`s non-viral platform incorporates a novel DNA construct called closed-ended DNA, or ceDNA; a unique cell-targeted lipid nanoparticle delivery system, or ctLNP; and a highly scalable capsid-free manufacturing process that uses its proprietary cell-free rapid enzymatic synthesis, or RES, to produce ceDNA. The platform is designed to enable multi-year durability from a single dose, to deliver large genetic payloads, including multiple genes, to specific tissues, and to allow titration and redosing to adjust or extend expression levels in each patient. RES has the potential to expand Generation Bio`s manufacturing scale to hundreds of millions of doses to support their mission to extend the reach of genetic medicine to more people, living with more diseases, around the world.
Born from the founders` recognition that their diverse but complementary skills and experiences would enable them to successfully address challenges that others had not, the company set about solving a vexing problem: how to bring true proteomics to the world in a way that accelerates therapeutic development, dramatically improves medical diagnostics, and makes personalized and predictive medicine a reality. The extraordinary team at Nautilus represents a wide spectrum of disciplines and expertise, including: protein chemists, chip designers, molecular biologists, data scientists, material scientists, biophysicists, optical engineers, microfluidics engineers, bioinformaticists, software engineers, and more. Nautilus is positioned to revolutionize proteomics, transform the way drugs are developed, and significantly improve the way human health is managed.
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