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YU1206

Development stage: Unknown
Modality: Small Molecules

Overview

**YU1206** is a novel small molecule degrader targeting LRF (Leukemia/Lymphoma-Related Factor, also known as ZBTB7A), a transcriptional repressor that inhibits fetal hemoglobin (HbF) expression in adult erythroid cells. By inducing LRF degradation, YU1206 promotes HbF upregulation, mimicking the mechanism of approved HbF inducers like hydroxyurea or BCL11A-targeting therapies used in sickle cell disease and beta-thalassemia. Preclinical data demonstrate effective HbF induction without significantly impairing erythroid maturation or peripheral blood counts, positioning it as a potential oral therapeutic for hemoglobinopathies.[7]

Targets

ZBTB7A (Zinc finger and BTB domain-containing protein 7A)

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