MiNA Therapeutics is launching the first saRNA human trial of its kind – OUTREACH for patients with severe liver cancer.
MiNA Therapeutics is a London based RNA specialist, developing RNA activation therapeutics that restore normal function to patients’ cells with an initial focus on severe liver cancer.
The study is the first-in-human trial of a small activating RNA (saRNA) and is designed to assess the safety and tolerability of their candidate MTL-CEBPA.
MTL-CEBPA consists of a double stranded RNA molecule, formulated into a ‘Smarticles‘ (liposomal nanoparticles) which are designed to activate the CEBPA gene.
The CEBPA gene encodes for the CCAAT/enhancer binding protein alpha (C/EBP-a), a transcription factor that acts as a master regulator of cell lineage determination and differentiation in several tissues including liver, myeloid cells and adipose tissue.
C/EBP-a plays an important role in normal liver function and the benefits of increasing its expression have been demonstrated in multiple pre-clinical models of disease.