MBX Biosciences, Inc. Common Stock
MBX Biosciences, Inc., a clinical-stage biopharmaceutical company, focuses on the discovery and development of precision peptide therapies for the treatment of endocrine and metabolic disorders. Its lead product candidate is MBX 2109, a parathyroid hormone peptide prodrug, which is in Phase 2 clinical trial for the treatment of chronic hypoparathyroidism. The company is also developing MBX 1416, a long-acting glucagon-like peptide-1 (GLP-1) receptor antagonist that is in Phase 1 clinical trial a potential therapy for post-bariatric hypoglycemia, a chronic complication of bariatric surgery. In addition, it is developing MBX 4291, a lead obesity product candidate, which is in investigational new drug-enabling studies for the treatment of obesity and co-morbidities. The company was founded in 2018 and is based in Carmel, Indiana.
Open MBX dashboard ↗Rocket Pharmaceuticals, Inc. Common Stock
Rocket Pharmaceuticals, Inc., together with its subsidiaries, operates as a late-stage biotechnology company that focuses on developing gene therapies for rare and devastating diseases in the United States. It develops vivo adeno-associated viral (AAV) programs, which include programs for Danon disease (DD), a multi-organ lysosomal-associated disorder leading to early death due to heart failure (RP-A501), which is in Phase 2 trial; Plakophilin-2 Arrhythmogenic Cardiomyopathy (PKP2-ACM), an inheritable cardiac disorder that is characterized by a progressive loss of cardiac muscle mass, severe right ventricular dilation, dysplasia, fibrofatty replacement of the myocardium and a high propensity to arrhythmias and sudden death (RP-A601), which is in phase 1; and BAG3 Dilated Cardiomyopathy (BAG3-DCM), which is a form of cardiomyopathy and is characterized by progressive thinning of the walls of the heart, which is in preclinical stage. It also develops vivo lentiviral (LV) programs, which include programs for Leukocyte Adhesion Deficiency-I (LAD-I), a genetic disorder that causes the immune system to malfunction (RP-L201); Fanconi Anemia (FA), a genetic defect in the bone marrow that reduces production of blood cells or promotes the production of faulty blood cells (RP-L102); and Pyruvate Kinase Deficiency (PKD), a red blood cell autosomal recessive disorder that results in chronic non-spherocytic hemolytic anemia (RP-L301). It has license agreements with Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas, Centro de Investigacion Biomedica En Red, and Fundacion Instituto de investigacion Sanitaria Fundacion Jimenez Diaz; UCL Business PLC; The Regents of the University of California; REGENXBIO, Inc.; and Temple University. Rocket Pharmaceuticals, Inc. was founded in 1999 and is headquartered in Cranbury, New Jersey.
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