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One Cell Systems is a Cambridge, MA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
At Travere Therapeutics, our mission is to identify, develop and deliver life-changing therapies to people living with rare disease. #InRareForLife
Tomtec Inc is a Hamden, CT-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
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.
Applied Molecular Therapeutics (AMT), a public biopharmaceutical company based in South San Francisco, California, is committed to developing novel, oral biological therapeutics to treat severe autoimmune, metabolic and inflammatory diseases. AMT is leveraging its propriety technology platforms to create gastrointestinal (GI)-select therapeutics that harness naturally occurring transport and targeting mechanisms to cross the protective barrier of the intestinal epithelium (IE). Once across the IE, the therapeutics gain privileged access to the immune cell-rich environment of the GI tissue as well as the hepatic portal system and downstream systemic circulation. With this privileged access, AMT`s goal is to develop transformative new oral therapeutic treatment options that offer patients greater efficacy and tolerability than available with today`s therapeutics. AMT has a robust pipeline of oral biologic product opportunities, including AMT-101 which is currently in Phase 1b clinical development for the treatment of adults with ulcerative colitis (UC). AMT-101 is a novel, gut-selective, investigational oral biologic fusion protein of interleukin 10 (IL-10), an anti-inflammatory cytokine. Derived from AMT`s proprietary platforms, AMT-101 is engineered to cross the selective barrier of the IE and enhance localized IL-10 directly within the immune-cell rich environment of GI tissue where up to 75 percent of immune cells reside. This direct targeting of the immune system creates the potential for AMT-101 to address, at its point of origin, the immune dysregulation that gives rise to UC and other inflammatory bowel diseases, and restore immune homeostasis with minimal systemic exposure and potentially fewer adverse events compared to systemic administration of IL-10. AMT has amassed a broad global IP portfolio surrounding its multiple technology platforms and therapeutic programs.