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Celula Inc is a San Diego, CA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Asklepios BioPharmaceutical, Inc. (AskBio) is a leading clinical-stage adeno-associated virus (AAV) gene therapy company dedicated to improving the lives of patients with rare diseases and other genetic disorders.
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.
There are approximately 7,000 rare disorders worldwide. Each day more are being discovered. 95% of rare diseases have not one single FDA approved treatment. Glycomine develops therapeutics for diseases which have no treatment options.
Nacalai USA is a San Diego, CA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.