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Crinetics Pharmaceuticals, Inc., a clinical-stage pharmaceutical company, focuses on the discovery, development, and commercialization of novel therapeutics for rare endocrine diseases and endocrine-related tumors. The company's lead product candidate is paltusotine, an oral selective nonpeptide somatostatin receptor type 2 agonist, which is in Phase 3 trial for the treatment of acromegaly; and Phase 2 trial for treating carcinoid syndrome associated with neuroendocrine tumors. It is also developing CRN04894, an investigational oral nonpeptide product candidate to antagonize the adrenocorticotrophic hormone (ACTH) receptor that has completed a Phase 1 study for the treatment of diseases caused by excess ACTH, including congenital adrenal hyperplasia and Cushing's disease. In addition, the company is developing antagonists of the parathyroid hormone (PTH) receptor for the treatment of primary hyperparathyroidism and humoral hypercalcemia of malignancy, and other diseases of excess PTH; identified investigational orally available somatostatin receptor type 3 targeted nonpeptide agonists for the treatment of autosomal dominant polycystic kidney disease; and developing thyroid-stimulating hormone receptor antagonists for the treatment of graves' disease and thyroid eye disease, as well as Oral GLP-1 and GIP nonpeptides for the treatment of diabetes and obesity. Crinetics Pharmaceuticals, Inc. was incorporated in 2008 and is headquartered in San Diego, California.

IVERIC bio, Inc., a biopharmaceutical company, focuses on the discovery and development of novel treatments for retinal diseases with unmet medical needs. The company develops Zimura, an inhibitor of complement factor C5 comprising GATHER1, which has completed Phase 2/3 clinical trial; GATHER2 that is in Phase 3 clinical trial for the treatment of geographic atrophy (GA) secondary to dry age-related macular degeneration (AMD); and STAR or OPH2005 that is in Phase 2b clinical trial for the treatment of autosomal recessive Stargardt disease. Its preclinical product candidatures include IC-500, a high temperature requirement A serine peptidase 1 protein inhibitors for the treatment of GA and other age-related retinal diseases. The company also develops OPH2001 that has completed Phase 1/2a clinical trial of various doses of avacincaptad pegol (ACP) administered as a monotherapy for the treatment of GA; OPH2000, which has completed Phase 1/2a clinical trial of various doses of ACP administered in combination with Lucentis for the treatment of wet AMD; OPH2007 that has completed Phase 2a clinical trial for the treatment of wet AMD; and OPH2002, which has completed Phase 2a clinical trial for the treatment of idiopathic polypoidal choroidal vasculopathy. In addition, its minigene programs comprises miniCEP290 program for LCA10; miniABCA4 Program for STGD1; and miniUSH2A Program for usher syndrome type 2A-Related inherited retinal diseases (IRDs). The company was formerly known as Ophthotech Corporation and changed its name to IVERIC bio, Inc. in April 2019. The company was incorporated in 2007 and is based in Parsippany, New Jersey. As of July 11, 2023, IVERIC bio, Inc. operates as a subsidiary of Astellas US Holding, Inc.

Magenta Therapeutics, Inc., a biotechnology company focus on improving stem cell transplantation. Its product candidates are designed to bring the curative power of stem cell transplant to patients with blood cancers, genetic diseases, and autoimmune diseases. The company's product portfolio includes MGTA-117, an anti-CD117 antibody conjugated to an amanitin payload that targets hematopoietic stem cells (HSCs) and leukemia cells; and MGTA-45, an anti-human CD45 antibody conjugated to a DNA-interacting payload for HSCs, leukemia cells, and immune cells. In addition, it focus on development of ADC-based conditioning program that targets a receptor of T cells; and novel conditioning and translation stem cell sciences, as well as develops cell therapy program, E478, which is a small molecule aryl hydrocarbon receptor antagonist for stem cell-based gene therapy and genome editing. In addition, the company has a research and clinical collaboration agreement with AVROBIO, Inc. for the treatment of lysosomal storage disorders; and Beam Therapeutics, Inc. to evaluate the potential utility of MGTA-117 for conditioning of patients with sickle cell disease and beta-thalassemia. Magenta Therapeutics, Inc. was formerly known as HSCTCo Therapeutics, Inc. and changed its name to Magenta Therapeutics, Inc. in February 2016. The company was incorporated in 2015 and is headquartered in Cambridge, Massachusetts.

Marinus Pharmaceuticals, Inc., a pharmaceutical company, focuses on development and commercialization of therapeutic products for patients suffering from rare genetic epilepsies and other seizure disorders. It offers ZTALMY (ganaxolone), an oral suspension for the treatment of seizures associated with cyclin-dependent kinase-like 5 deficiency disorder for adult and pediatric patient populations in acute and chronic care, and in-patient and self-administered settings. The company's ZTALMY product candidate acts at synaptic and extrasynaptic GABAA receptors, a target for its anti-seizure, antidepressant, and anxiolytic potential. It is developing ganaxolone for treating genetic epilepsy disorders, such as PCDH19-related epilepsy, and tuberous sclerosis complex. Marinus Pharmaceuticals, Inc. has license agreement with Purdue Neuroscience Company and CyDex Pharmaceuticals, Inc.; and collaboration agreement with Orion Corporation and Tenacia Biotechnology (Shanghai) Co., Ltd. Marinus Pharmaceuticals, Inc. was incorporated in 2003 and is headquartered in Radnor, Pennsylvania.

Neoleukin Therapeutics, Inc., a biopharmaceutical company, develops immunotherapies for cancer, inflammation, and autoimmunity disorders using protein design technology. The company's lead product candidate is NL-201, a de novo protein designed to mimic the therapeutic activity of the cytokines interleukin (IL)-2/IL-15 for the treatment of various types of cancer, including renal cell carcinoma and melanoma. The company was formerly known as Aquinox Pharmaceuticals, Inc. and changed its name to Neoleukin Therapeutics, Inc. in August 2019. Neoleukin Therapeutics, Inc. was founded in 2003 and is headquartered in Seattle, Washington.