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XPACE-1001 is a preclinical-stage therapeutic candidate developed by Xanadu Bio targeting **CFTR** (cystic fibrosis transmembrane conductance regulator) for the treatment of **cystic fibrosis**. It functions as a **CFTR modulator**, a class of agents designed to correct the defective CFTR protein underlying the disease, potentially restoring chloride ion transport across epithelial cell membranes to alleviate mucus buildup in the lungs and other organs. Xanadu Bio's pipeline emphasizes mRNA-based technologies, though specific details on XPACE-1001's formulation remain undisclosed.[1]
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