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Enzyvant is also advancing the development of RVT-802, an investigational tissue-based biologic therapy for the potential treatment of primary immune deficiency associated with complete DiGeorge Syndrome. RVT-802 has been granted orphan drug designation, Breakthrough Therapy designation, Regenerative Medicine Advanced Therapy designation, and pediatric rare disease designation by the U.S. Food and Drug Administration. Enzyvant anticipates a potential BLA filing for RVT-802 in the first half of 2018. Enzyvant plans to develop treatments for additional rare diseases with high unmet need.
Kansas Bioscience Organization is a Overland Park, KS-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Acceleron Pharma, Inc. is an American clinical stage biopharmaceutical company based in Cambridge, Massachusetts with a broad focus on developing medicines that regulate the transforming growth factor beta (TGF-β) superfamily of proteins, which play fundamental roles in the growth and repair of cells and tissues such as red blood cells, muscle, bone, and blood vessels.
Banyan Biomarkers Inc is a Alachua, FL-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Crinetics Pharmaceuticals is a clinical stage pharmaceutical company focused on the discovery, development, and commercialization of novel therapeutics for rare endocrine diseases and endocrine-related tumors. The company`s lead product candidate, paltusotine (formerly CRN00808), is an investigational, oral, selective nonpeptide somatostatin receptor type 2 biased agonist for the treatment of acromegaly, an orphan disease affecting more than 26,000 people in the United States. A Phase 3 program in acromegaly with paltusotine is underway. Crinetics also plans to advance paltusotine into a Phase 2 trial for the treatment of carcinoid syndrome associated with neuroendocrine tumors. The company is also developing CRN04777, an investigational, oral, nonpeptide somatostatin receptor type 5 (SST5) agonist for congenital hyperinsulinism, as well as CRN04894, an investigational, oral, nonpeptide ACTH antagonist for the treatment of Cushing`s disease, congenital adrenal hyperplasia and other diseases of excess ACTH. All of the company`s drug candidates are new chemical entities resulting from in-house drug discovery efforts and are wholly owned by the company.