Amylyx Pharmaceuticals, Inc. Common Stock
Amylyx Pharmaceuticals, Inc., a clinical-stage pharmaceutical company, engages in the discovery and development of treatment options for neurodegenerative diseases and endocrine conditions in the United States. Its lead products candidate includes avexitide, an investigational, first-in-class glucagon-like peptide-1, or GLP-1 receptor antagonist, which is in phase 3 clinical trial to treat post-bariatric hypoglycemia and congenital hyperinsulinism. The company also develops AMX0035, an oral, fixed-dose combination of sodium phenylbutyrate and taurursodiol which is phase 2 clinical trial for the treatment of wolfram syndrome, as well as in phase 2b/3 clinical trial to treat progressive supranuclear palsy; AMX0114, which is in phase 1 clinical trial for the treatment of amyotrophic lateral sclerosis; and long-acting GLP-1 receptor antagonist to treat post-bariatric hypoglycemia and other rare diseases. Amylyx Pharmaceuticals, Inc. was founded in 2013 and is headquartered in Cambridge, Massachusetts.
Open AMLX 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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