Moleculin Biotech, Inc.
Moleculin Biotech, Inc., a late-stage pharmaceutical development company, engages in the development of therapeutic candidates for the treatment of tumors and viruses. Its lead drug candidate is Annamycin, an anthracycline, which is in clinical stage for the treatment of relapsed or refractory acute myeloid leukemia and soft tissue sarcoma lung metastases. The company is also developing WP1066 portfolio, including WP1066, WP1193, and WP1220, which are immune/transcription modulators designed to inhibit phosphorylated signal transducer and activator of transcription for brain tumors and pancreatic and other cancers. In addition, it is developing WP1122 portfolio, such as WP1122, WP1096, and WP1097 designed to exploit the uses of inhibitors of glycolysis comprising 2-deoxy-D-glucose for the treatment of pathogenic viruses and various cancers. The company was incorporated in 2015 and is headquartered in Houston, Texas.
Open MBRX 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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