Design Therapeutics, Inc. Common Stock
Design Therapeutics, Inc. a clinical-stage biopharmaceutical company, research, designs, develops, and commercializes small molecule therapeutic drugs for the treatment of genetic diseases in the United States. The company utilizes its GeneTAC platform to design and develop therapeutic candidates for inherited diseases caused by nucleotide repeat expansion. Its lead product candidates for potentially disease-modifying treatment comprises Friedreich Ataxia, a monogenic, autosomal recessive, progressive multi-system disease that affects organ systems dependent on mitochondrial function that brings to neurological, cardiac, and metabolic dysfunction; Myotonic Dystrophy Type-1, a dominantly-inherited, monogenic progressive neuromuscular disease affecting skeletal muscle, heart, brain, and other organs; Fuchs Endothelial Corneal Dystrophy, a genetic eye disease characterized by bilateral degeneration of corneal endothelial cells and progressive loss of vision; and Huntington's Disease, a dominantly inherited, monogenic neurodegenerative disease characterized by movement, cognitive, and psychiatric disorders. Design Therapeutics, Inc. was incorporated in 2017 and is headquartered in Carlsbad, California.
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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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