Cogent Biosciences, Inc. Common Stock
Cogent Biosciences, Inc., a biotechnology company, focuses on developing precision therapies for genetically defined diseases. Its lead product candidate includes bezuclastinib (CGT9486), a selective tyrosine kinase inhibitor in Phase 3 trial designed to target mutations within the KIT receptor tyrosine kinase, including KIT D816V mutation that drives systemic mastocytosis, as well as other mutations in KIT exon 17, which are found in patients with advanced gastrointestinal stromal tumors. The company also develops CGT4859, a reversible and selective fibroblast growth factor receptor 2 inhibitor in Phase 1 trial for patients with documented FGFR mutations, including advanced cholangiocarcinoma. It has a licensing agreement with Plexxikon Inc. for the research, development, and commercialization of bezuclastinib. Cogent Biosciences, Inc. is headquartered in Waltham, Massachusetts.
Open COGT dashboard ↗Ultragenyx Pharmaceutical Inc.
Ultragenyx Pharmaceutical Inc., a biopharmaceutical company, focuses on the identification, acquisition, development, and commercialization of novel products for the treatment of rare and ultra-rare genetic diseases in North America, Latin America, Europe, the Middle East, Africa, and the Asia-Pacific. Its biologic products include Crysvita (burosumab), an antibody targeting fibroblast growth factor 23 for the treatment of X-linked hypophosphatemia, as well as tumor-induced osteomalacia; Mepsevii, an enzyme replacement therapy for the treatment of children and adults with Mucopolysaccharidosis VII; Dojolvi for the treatment of long-chain fatty acid oxidation disorders; and Evkeeza (evinacumab) for the treatment of homozygous familial hypercholesterolemia. The company's products candidates that are in Phase 3 clinical trials include UX143, a human monoclonal antibody for the treatment of osteogenesis imperfecta; UX111, an AAV9 gene therapy product candidate for the treatment of patients with Sanfilippo syndrome type A, or MPS IIIA, a rare lysosomal storage disease; DTX401, an adeno-associated virus 8 (AAV8) gene therapy clinical candidate for the treatment of patients with glycogen storage disease type Ia; DTX301, an AAV8 gene therapy for the treatment of patients with ornithine transcarbamylase; and GTX-102, an antisense oligonucleotide for the treatment of Angelman syndrome. It also develops UX701 that is in Phase 2 clinical trial for the treatment of Wilson disease; and UX053 for the treatment of glycogen storage disease type III. Ultragenyx Pharmaceutical Inc. has collaboration and license agreement with Kyowa Kirin Co., Ltd.; Saint Louis University; Baylor Research Institute; REGENXBIO Inc.; GeneTx; Mereo; University of Pennsylvania; Solid Biosciences Inc.; Regeneron; Abeona; and Arcturus Therapeutics Holdings Inc. The company was incorporated in 2010 and is headquartered in Novato, California.
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