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IQuum is a Marlborough, MA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
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.
Sengenics is a functional proteomics company that leverages its patented KREX technology to discover autoantibody biomarker signatures for prediction of drug response and severe immune-related adverse events (irAEs). KREX™ was invented and patented by Professor Jonathan Blackburn whilst he was a member of the faculty at the University of Cambridge. Jonathan is the CSO of Sengenics. The key application of KREX™ is the discovery of autoantibody biomarker signatures for prediction of drug response and severe immune-related adverse events (irAEs). KREX™ can also be used to identify autoantibody biomarkers that may be used to diagnose cancer, autoimmune or neurodegenerative conditions years before conventional diagnostic tests. Some autoantibodies that are identified as diagnostic biomarkers may be protective and have potential in themselves as therapeutic biomolecules.
GTx, Inc. is one of the leading companies in the Healthcare, Pharmaceuticals, and Biotech sector.
Castle Creek Biosciences, Inc. is a clinical-stage cell and gene therapy company focused on developing and commercializing disease-modifying therapies for patients suffering from rare diseases for which there is a lack of available treatment options. The company`s proprietary autologous fibroblast platform potentially allows for the development of personalized, targeted and redosable cell-based gene therapy product candidates for monogenic and chronic disorders. The company`s most advanced product candidate, dabocemagene autoficel (D-Fi), is currently being evaluated in a Phase 3 clinical trial for the localized treatment of chronic wounds due to recessive dystrophic epidermolysis bullosa (RDEB). The company is also currently evaluating FCX-013 in a Phase 1/2 clinical trial for the treatment of moderate to severe localized scleroderma. In addition, Castle Creek Biosciences is pursuing discovery and potential development of early-stage novel product candidates with the goal of expanding its robust pipeline into other rare diseases and broader indications where there are significant unmet needs. The company operates an in-house, commercial-scale manufacturing facility in Exton, Pennsylvania that benefits from the validated systems and processes previously implemented at the site for manufacture of an FDA-approved cell therapy product. Castle Creek Biosciences, Inc. is a portfolio company of Paragon Biosciences, LLC.