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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.
Bioverativ Inc. is an American multinational biotechnology that specializes in the discovery, development, and delivery of therapies for the treatment of haemophilia.
insitro is a data-driven drug discovery and development company that leverages machine learning and high-throughput biology to transform the way medicines are created to help patients. At insitro, we are rethinking the entire drug discovery process, from the perspective of machine learning, human genetics, and high-throughput, quantitative biology. Over the past five decades, we have seen the development of new medicines becoming increasingly more difficult and expensive, leaving many patients with significant unmet need. We`re embarking on a new approach to drug development – one that leverages machine learning and unique in vitro strategies for modeling disease state and designing new therapeutic interventions. We aim to eliminate key bottlenecks in traditional drug discovery, so we can help more people sooner and at a much lower cost to the patient and the healthcare industry. We believe that by harnessing the power of technology to interrogate and measure human biology, we can have a major impact on many diseases. We invest heavily in cutting edge bioengineering technologies to enable the construction of large-scale, high-quality data sets that are designed specifically to drive machine learning methods. Our first application is to use human genetics, functional genomics, and machine learning to build a new generation of in vitro human cell-derived disease models whose response to perturbation is designed to be predictive of human clinical outcomes.
Peplin Limited(Now LeoPharma) is a Emeryville, CA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Perimeter Resources is a Houston, TX-based company in the Healthcare, Pharmaceuticals, and Biotech sector.