Aptevo Therapeutics Inc
Aptevo Therapeutics Inc., a clinical-stage research and development biotechnology company, focuses on developing immunotherapeutic candidates for the treatment of various forms of cancer in the United States. The company develops its products using ADAPTIR and ADAPTIR-FLEX platforms to generate monospecific, bispecific, and multi-specific antibody candidates capable of enhancing the human immune system against cancer cells. Its lead clinical blood cancer candidate is mipletamig that is in Phase 1b/2 clinical trial for the treatment of acute myelogenous leukemia. The company also develops ALG.APV-527, a novel investigational bispecific ADAPTIR candidate that is in Phase 1 clinical trial for the treatment of non-small cell lung cancer, head and neck, colorectal, pancreatic, breast, and other solid tumors; APVO603, a preclinical dual agonist bispecific ADAPTIR candidate to treat multiple solid tumors; APVO442, a preclinical bispecific candidate based on the ADAPTIR-FLEX platform technology for the treatment of multiple solid tumors; and APVO711, a preclinical dual mechanism bispecific ADAPTIR candidate to treat prostate cancer. It has a collaboration and option agreement with Alligator Bioscience AB to develop ALG.APV-527. Aptevo Therapeutics Inc. was incorporated in 2016 and is headquartered in Seattle, Washington.
Open APVO 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.
Open RARE dashboard ↗