MTL-CEBPA

MTL-CEBPA is a first-in-class small activating RNA (saRNA) oligonucleotide drug designed to upregulate the transcription factor CCAAT/enhancer-binding protein alpha (C/EBPα). This transcription factor acts as a master regulator of hepatic and myeloid cell functions and is involved in multiple oncogenic processes. By specifically targeting the promoter region of the CEBPA gene, MTL-CEBPA restores its transcription, leading to increased mRNA and protein expression. This activation reduces immune suppression by myeloid cells within the tumor microenvironment, thereby enhancing anti-tumor immune responses and potentially improving the efficacy of other cancer therapies such as tyrosine kinase inhibitors or immune checkpoint inhibitors. The drug is formulated using SMARTICLES liposomal technology for efficient delivery and stability. It has demonstrated anti-inflammatory and anti-cancer activity in preclinical models and early-phase clinical trials for advanced liver cancers including hepatocellular carcinoma[1][3][4][5][6].

Other names

CEBPA-targeting saRNA MTL-CEBPA liposome