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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Telix Pharmaceuticals Limited, a commercial-stage biopharmaceutical company, focuses on the development and commercialization of therapeutic and diagnostic radiopharmaceuticals. The company operates through three segments: Precision Medicine, Therapeutics, and Manufacturing Solutions. Its lead therapeutic product candidate is TLX591, a lutetium-labeled radio antibody-drug conjugate (rADC), which is in a Phase 3 clinical trial in patients with advanced prostate cancer. The company develops TLX250, an rADC for treating advanced metastatic kidney cancer; TLX101, a systemic therapy for the treatment of glioblastoma; TLX66, a product candidate for bone marrow conditioning for hematopoietic stem cell transplant conditioning; and Illuccix and Gozellix for the treatment of prostate cancer. The company is also developing TLX592, a prostate cancer therapy candidate for targeted alpha therapy based on its proprietary RADmAb-engineered antibody technology; TLX252 for the treatment of patients with advanced metastatic kidney cancer; TLX400, a bladder fibroblast activation protein for treating various tumors; TLX102, a large amino acid transporter-targeting small molecule-based alpha therapy candidate for the treatment of glioblastoma and multiple myeloma; TLX300 for treating soft tissue sarcoma; and TLX090, a bone-seeking agent for bone metastases and pain palliation. In addition, it is developing TLX250-CDx, a positron emission tomography (PET) diagnostic imaging agent for the characterization of renal masses as clear cell renal cell carcinoma; TLX101-CDx, a PET diagnostic agent to image cancerous lesions in the brain; and TLX66-CDx, a cluster of differentiation 66 imaging agent for osteomyelitis (bone infection). It operates in Australia, Belgium, Canada, the United Kingdom, the United States, and internationally. The company was founded in 2015 and is headquartered in North Melbourne, Australia.
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