uniQure N.V.
uniQure N.V. develops treatments for patients suffering from rare and other devastating diseases in the United States. The company offers HEMGENIX that allows people living with hemophilia B to produce factor IX, which can lower the risk of bleeding. Its lead product candidate is AMT-130, a gene therapy candidate, which is in Phase I/II clinical study for the treatment of Huntington's disease. The company also develops AMT-260, which is in Phase I/IIa clinical trial for the treatment of mesial temporal lobe epilepsy; AMT-162, which is in Phase I/IIa clinical trial to treat superoxide dismutase enzyme-amyotrophic lateral sclerosis; and AMT-191, an investigational gene therapy candidate which is in phase I/IIa clinical trial for the treatment of fabry disease. It has a licensing agreement with Apic Bio to develop, manufacture, and commercialize intrathecally administered investigational gene therapy for ALS caused by mutations in SOD-1; and development and commercial supply agreement with CLS Bhering. uniQure N.V. was founded in 1998 and is headquartered in Amsterdam, the Netherlands.
Open QURE 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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