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 ↗X4 Pharmaceuticals, Inc. Common Stock
X4 Pharmaceuticals, Inc., a biopharmaceutical company, focuses on the research, development, and commercialization of novel therapeutics for the treatment of rare diseases of the immune system. It offers XOLREMDI, an orally bioavailable selective antagonist of chemokine receptor C-X-C chemokine receptor type 4 (CXCR4) for the treatment of patients with warts, hypogammaglobulinemia, infections, and myelokathexis syndrome. The company also develops mavorixafor that is in Phase 3 4WARD clinical trial to evaluate the efficacy, safety, and tolerability of oral, once-daily mavorixafor with or without stable doses of G-CSF in people with congenital, acquired primary autoimmune, or idiopathic CN who are experiencing recurrent and/or serious infections. It has a license agreement with Abbisko Therapeutics Co Ltd. to develop, manufacture, and commercialize mavorixafor in combination with checkpoint inhibitors or other agents in oncology indications; and with Norgine to develop, manufacture, and commercialize mavorixafor in Europe, Australia, and New Zealand. The company is headquartered in Boston, Massachusetts.
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