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Cemsidomide is an orally bioavailable small molecule that acts as a modulator of the E3 ubiquitin ligase complex containing cereblon (CRL4–CRBN E3 ubiquitin ligase). It is designed to be a highly potent and selective degrader of the transcription factors Ikaros (IKZF1) and Aiolos (IKZF3), which are key regulators in hematopoietic cell differentiation. By binding to cereblon, cemsidomide alters the activity of the E3 ubiquitin ligase, leading to targeted ubiquitination and proteasome-mediated degradation of IKZF1/IKZF3. This results in downregulation of IRF4, another transcription factor critical for survival of multiple myeloma and lymphoma cells, thereby exerting immunomodulatory and antineoplastic effects. Cemsidomide is being developed primarily for relapsed/refractory multiple myeloma and non-Hodgkin’s lymphoma[1][2][4][6][8].
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