WIZ/ZBTB7A Degrader BMS-986470

**Semantic type:** Pharmacologic Substance

**Definition:** A modulator of the E3 ubiquitin ligase complex containing cereblon (CRL4-CRBN E3 ubiquitin ligase) and targeted degrader of both human zinc finger and BTB domain-containing protein 7A (ZBTB7A) and human widely interspaced zinc finger protein (WIZ), with potential use in the treatment of sickle cell disease (SCD). Upon oral administration, WIZ/ZBTB7A degrader BMS-986470 specifically targets and binds to cereblon (CRBN), a component of the CRL4-CRBN E3 ubiquitin ligase complex. This catalyzes ubiquitination and proteasome-mediated degradation of WIZ and ZBTB7A. Degradation of WIZ and ZBTB7A decreases gamma-globin silencing, thereby increasing gamma-globin expression. This may increase the level of fetal hemoglobin (HbF), which counteracts sickle cell hemoglobin (HbS) polymerization and reduces red blood cell (RBC) sickling and damage.

**Synonyms:** - BMS 986470 - BMS-986470 - BMS986470 - Fetal Hemoglobin Activating Cereblon E3 Ligase Modulating Drug BMS-986470 - HbF-activating CELMoD BMS-986470 - ZBTB7A/WIZ Degrader BMS-986470 - ZBTB7A/WIZ Dual Degrader BMS-986470