Kazia Therapeutics Limited American Depositary Shares
Kazia Therapeutics Limited, together with its subsidiaries, operates as an oncology-focused biotechnology company in South Korea. Its lead development candidate is paxalisib, a small molecule, brain-penetrant inhibitor of the phosphoinositide-3-kinase (PI3K)/AKT/mammalian target of rapamycin (mTOR) pathway, which is in Phase 3 clinical trial, for the treatment of glioblastoma, isocitrate dehydrogenase-mutant glioma, diffuse intrinsic pontine glioma, atypical teratoid/rhabdoid tumor, brain metastases, primary central nervous system (CNS) lymphoma, triple negative breast cancer, and ovarian cancer. The company also develops EVT801, a small-molecule selective inhibitor of vascular endothelial growth factor receptor 3, which is in Phase 1 clinical trial, to treat advanced solid tumors. It has collaborations with the Australian and New Zealand Children's Haematology/Oncology Group, OIMR Berghofer Medical Research Institute, Genentech Inc., Global Coalition for Adaptive Research, Vivesto AB, Simcere Pharmaceutical Group Ltd, Evotec SE, and Sovargen Co., Ltd. The company was formerly known as Novogen Limited and changed its name to Kazia Therapeutics Limited in November 2017. Kazia Therapeutics Limited was incorporated in 1994 and is based in Sydney, Australia.
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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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