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Latozinemab (AL001, GSK4527223) is a recombinant human monoclonal antibody developed to treat neurodegenerative diseases associated with progranulin deficiency. Its primary mechanism of action is the inhibition of sortilin (SORT1), a receptor that mediates the degradation of progranulin (PGRN). By blocking sortilin, latozinemab increases circulating and central nervous system levels of PGRN—a key regulator of immune activity in the brain implicated in frontotemporal dementia (FTD), Alzheimer’s disease, and Parkinson’s disease. Latozinemab has received Breakthrough Therapy Designation and Orphan Drug Designation from the FDA for frontotemporal dementia due to a progranulin gene mutation (FTD-GRN) and is currently being evaluated in Phase 3 clinical trials for this indication. The drug is administered via intravenous infusion[1][2][3][4][5][6].
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