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NXC-201 is a sterically-optimized, B-cell maturation antigen (BCMA)-targeted chimeric antigen receptor T (CAR-T) cell therapy. It is being developed for the treatment of relapsed or refractory light chain (AL) amyloidosis and multiple myeloma, with potential expansion into autoimmune diseases such as systemic lupus erythematosus, myasthenia gravis, and multiple sclerosis. NXC-201 works by engineering a patient’s own T cells to express a CAR that specifically targets BCMA on malignant plasma cells. This leads to targeted destruction of these cells upon reinfusion into the patient. The therapy has demonstrated high response rates and an absence of neurotoxicity in clinical trials to date. NXC-201 has received Orphan Drug Designation from both the FDA and EMA for AL amyloidosis and multiple myeloma, as well as Regenerative Medicine Advanced Therapy designation from the FDA for AL amyloidosis[1][4][5][6][7].
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