c-Myb mRNA degrader RGT-61159

An orally bioavailable, small molecule mRNA modulator of the proto-oncogene c-Myb, with potential antineoplastic activity. Upon oral administration, c-Myb mRNA degrader RGT-61159 targets c-Myb RNA and selectively induces the inclusion of the cryptic "poison" exon into the c-Myb RNA transcript, resulting in the elimination of c-Myb mRNA transcript. This inhibits the production of the c-Myb protein. Suppression of c-Myb expression results in the restoration of normal differentiation pathways, increased antiproliferative effects, and the induction of apoptosis in tumor cells that overexpress c-Myb. c-Myb, an oncogenic transcription factor that plays a key role in the regulation of hematopoietic cell differentiation and proliferation, is dysregulated or overexpressed in a variety of tumor cell types.