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X4 Pharmaceuticals Inc is a late-stage clinical biopharmaceutical company engaged in the research and development of novel therapeutics for the treatment of rare diseases and those with limited treatment options, with a focus on conditions resulting from dysfunction of the immune system. The Company's lead clinical candidate is mavorixafor, a small molecule antagonist of the chemokine receptor CXCR4 that is being developed as an oral, once-daily therapy. It has the ability to increase the mobilization of mature, functional white blood cells from the bone marrow into the bloodstream. It also has two pre-clinical candidates X4P-003 and X4P-002 which are CXCR4 antagonists with different properties.
Editas Medicine Inc is a clinical-stage genome editing company dedicated to developing potentially transformative genomic medicines to treat a broad range of serious diseases. The company focuses on developing a proprietary genome editing platform based on clustered, regularly interspaced short palindromic repeats (CRISPR)/ CRISPR-associated protein 9 (Cas9 technology). It has developed CRISPR/Cas9 technology which uses a protein-RNA (ribonucleic acid) complex composed of the Cas9 enzyme bound to a guide RNA molecule designed to recognize a DNA (deoxyribonucleic acid) sequence that requires repair. The company is engaged in developing and commercializing genome editing technology.