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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.
Galenea Corp is a Cambridge, MA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Synageva BioPharma Corp. is a Lexington, MA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Eidos Therapeutics, a subsidiary of BridgeBio Pharma, is developing AG10 as a targeted therapeutic for transthyretin amyloidosis.
DBV Technologies (DBV) is a clinical-stage biopharmaceutical company focused on discovering safe and effective treatments for patients with food allergies and other unmet medical needs. DBV has developed a proprietary technology platform called Viaskin®, which is based on epicutaneous immunotherapy, or EPIT®, DBV`s method of delivering biologically active compounds to the immune system. This novel mechanism of action accesses the immune system through intact skin without allowing passage of the antigen into the bloodstream. The company is also exploring Viaskin® in other areas of unmet need including vaccines, inflammatory conditions and autoimmune diseases. DBV was founded in 2002 by Dr. Pierre-Henri Benhamou, Chairman and CEO, Dr. Christophe Dupont, Chairman of DBV`s Scientific Advisory Board, and Mr. Bertrand Dupont, Chief Technology Officer.