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Q1 2024

X4 Pharmaceuticals
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Q1 2024

7 May, 2024
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FDA Announcement

29 Apr, 2024
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X4 Pharmaceuticals, Inc., a late-stage clinical biopharmaceutical company, focuses on the research, development, and commercialization of novel therapeutics for the treatment of rare diseases. The company's lead product candidate is X4P-001, an oral medication targeted at patients with paroxysmal nocturnal hemoglobinuria, a life-threatening disorder characterized by blood clotting in the bone marrow causing severe anemia and life-threatening bleeding episodes. It also has a preclinical program focused on targeting complement inhibitor molecule C5 to treat acute liver failure that is currently in Phase I/II clinical trials.