Denali Therapeutics Inc. Common Stock
Denali Therapeutics Inc., a biopharmaceutical company, discovers and develops therapeutics to treat neurodegenerative and lysosomal storage diseases. The company develops Eclitasertib (SAR443122/DNL758) RIPK1 inhibitor program for peripheral inflammatory diseases; DNL343 eIF2B activator program for amyotrophic lateral sclerosis; BIIB122/DNL151 LRRK2 inhibitor program for Parkinson's disease; TAK-594/DNL593 program for frontotemporal dementia-granulin; DNL126 program for MPS IIIA (Sanfilippo Syndrome A); and DNL310 Tividenofusp alfa, an enzyme replacement therapy program for MPS II (Hunter Syndrome). Its Enzyme TransportVehicle (TV) programs include DNL952 for the treatment of Pompe disease; DNL111 for Parkinson's and Gaucher diseases; and DNL622 for Hurler syndrome (MPS I). The company's OTV programs also comprise DNL628 targeting tau for Alzheimer's disease; and DNL422 targeting alpha synuclein for Parkinson's disease, as well as antibody TV program includes DNL921 targeting Abeta for treating Alzheimer's disease. The company was formerly known as SPR Pharma Inc. and changed its name to Denali Therapeutics Inc. in March 2015. Denali Therapeutics Inc. was incorporated in 2013 and is headquartered in South San Francisco, California.
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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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