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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.
We are a biotechnology company integrating AI, computation and biological automation to accelerate the design of small molecule and protein therapeutics. Our mission is to decrease the timeline and cost of drug development, while improving the success rate of bring innovative medicines to patients in need.
Clinical stage gene therapy company using a constellation of proprietary AAV vector technologies to develop novel treatments for central nervous system and lysosomal storage diseases
AmProx is a Carlsbad, CA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
The Boston Biomedical Research Institute (BBRI) is dedicated to basic biomedical research to promote the understanding, treatment and prevention of specific human diseases. The areas of investigation concern the structure and function of muscle proteins,