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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.
GBT is a clinical-stage biopharmaceutical company determined to discover, develop and deliver innovative treatments that provide hope to underserved patient communities. GBT is developing its late-stage product candidate, voxelotor (previously called GBT440), for the treatment of sickle cell disease.
We are harnessing the #gut brain axis & the #CNS to develop a new class of therapeutics for people with neurological diseases & disorders
Viracta is focused on advancing a proprietary viral gene activation therapy to address cancers associated with the Epstein-Barr Virus, and will pursue opportunities to address other serious virus-associated disease.
Bonna-Agela Technologies is a Wilmington, DE-based company in the Healthcare, Pharmaceuticals, and Biotech sector.