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Taysha Gene Therapies is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we hope to rapidly translate our treatments from bench to bedside. We have combined our team`s proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to quickly and efficiently build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platform—an engine for new cures—to dramatically improve patients` lives.
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.
Rgenta Therapeutics is developing a pipeline of oral, small-molecule RNA-targeting medicines with an initial focus on oncology and neurological disorders. Our proprietary platform mines the massive genomics data to identify targetable RNA processing events and design small-molecule glues to modulate the interactions among the spliceosome, regulatory proteins, and RNAs
Raptor is a NASDAQ-listed biopharmaceutical company with the mission to develop and commercialize life-altering therapeutics that treat debilitating and often fatal diseases. Raptor`s lead product, PROCYSBI (cysteamine bitartrate) delayed-release capsules, received FDA approval on April 30, 2013. The Company anticipates receiving a determination from the EMA regarding its PROCYSBI marketing application in the European Union in the second half of 2013. Raptor’s product pipeline may advance therapeutic options for additional indications. Raptor`s management team consists of repeat entrepreneurs with the experience to build a successful company.
NantKwest is a pioneering, next generation, clinical-stage immunotherapy company focused on harnessing the unique power of our immune system using natural killer (NK) cells to treat cancer, infectious diseases and inflammatory diseases. NK cells are the body’s first line of defense due to the innate ability of NK cells to rapidly identify and destroy cells under stress, such as cancer or virally-infected cells.