Discovery & Optimization of First-in-Class Molecular Glue Degraders of the WIZ Transcription Factor for Fetal Hemoglobin Induction to Treat Sickle Cell Disease
Time: 11:30 am
day: Focus Day
Details:
- A phenotypic screen identified inducers of fetal hemoglobin to treat SCD
- Subsequent target elucidation identified CRBN dependent degradation of the transcription factor WIZ as driver of HbF induction
- Med chem optimization resulted in molecules with improved WIZ degradation selectivity and in vivo PK/PD and efficacy and candidates for development
