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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.
Keros Therapeutics is dedicated to the discovery and development of novel therapeutics for neuromuscular diseases
At GlycoMimetics, we are committed to helping to make a difference in the lives of people living with sickle cell, cancer, and other serious diseases. Our mission is to continue to advance our research and clinical candidates, providing hope for patients as we strive for tomorrow`s breakthrough treatments. GlycoMimetics is a clinical-stage biotechnology company. We use novel and proprietary glycobiology technology to develop treatments for diseases, particularly where there is high unmet need. Since the company`s inception in 2003, we have developed a robust, diversified product pipeline. We are a public company traded on the NASDAQ under the symbol GLYC.
We are pioneering a better drug discovery process using systems biology to improve the lives of people with neurodegenerative disorders.
Ansata Therapeutics is a La Jolla, CA-based company in the Healthcare, Pharmaceuticals, & Biotech sector.