Actinium Pharmaceuticals, Inc
Actinium Pharmaceuticals, Inc. develops antibody radiation conjugates and other targeted radiotherapies intended to improve outcomes for people who have failed existing oncology therapies. The company advances two clinical stage product candidates, Actimab-A, to treat patients with myeloid malignancies including acute myeloid leukemia and myelodysplastic syndromes; and Iomab-ACT, to improve patient access to and outcomes with cellular therapies, such as CAR-T for various blood cancer indications and gene therapies for non-malignant hematologic disorders such as sickle cell disease. It also develops ATNM-400, a preclinical non-prostate specific-membrane antigen targeting radiotherapy utilizing the Actinium-225 radioisotope for patients with prostate cancer. In addition, the company's Iomab-B is an induction and conditioning agent prior to bone marrow transplant in patients with relapsed and refractory acute myeloid leukemia (r/r AML). Further, it engages with the National Cancer Institute under the cooperative research and development agreement for the development of Actimab-A in AML and other myeloid malignancies, and its research and development activities primarily focus on advancing various preclinical programs for solid tumor indications. The company is based in New York, New York.
Open ATNM 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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