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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.
SalioGen Therapeutics is advancing the Exact DNA Integration Technology (EDIT) platform, the only genome engineering technology that is mammal-derived, for use in potentially curative non-viral gene therapy.
Pionyr Immunotherapeutics discovers and develops first-in-class biologic drugs that fine-tune natural immune responses to increase immune defenses for diseases such as cancer, or to dampen immune responses in autoimmune diseases.
KBI BioPharma is a Durham, NC-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
GTC Biotherapeutics is a Framingham, MA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.