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Sigilon Therapeutics’ mission is to create immune-protected, engineered human cells that restore normal physiology in a wide range of diseases without generating fibrosis or immune rejection, liberating patients from challenges associated with serious chronic diseases. We're based in Cambridge, Mass.
Cortendo AB is a global biopharmaceutical company founded in 1996, incorporated in Sweden, and based in the United States. Cortendo recognizes the urgent need to make new medicines available for people with orphan diseases, and the Company is committed to delivering therapies that make a difference. An orphan disease is one for which the pharmaceutical industry has not worked to make new medicines. It may be a rare disease (in the U.S. this is defined as a disease that affects fewer than 200,000 people) or a disease such as tuberculosis, cholera, typhoid or malaria that is not often diagnosed in developed countries but remains common in countries that are still developing. Cortendo’s initial strategic goal is to be the global leader in finding, developing and making medicines for people with orphan endocrine diseases, with its most advanced program in Cushing’s syndrome. Cortendo research led to the development of COR-003 (levoketoconazole) which is currently being studied in the Phase 3 global SONICS trial for the treatment of endogenous Cushing’s syndrome. COR-003 has received orphan designation from both the European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA). Cortendo is building the capabilities and resources to independently develop and commercialize its orphan assets in key global markets and to partner non-strategic product opportunities, such as BioPancreate-2001 for Type 1 and Type 2 diabetes. The company also intends to leverage its commercial expertise by working with partners to acquire, develop, and commercialize late-stage or commercial assets in a select few orphan disease focus areas.
moksha8 Pharmaceuticals is a Wayne, PA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Kira Pharmaceuticals is pioneering complement drug discovery to deliver transformative therapies for people with complement-mediated diseases
MBX Biosciences is a preclinical-stage biotechnology company using validated molecular targets to create medicines for rare endocrine diseases where current therapies are inadequate.