RNA therapy starts first-of-a-kind trial to treat Liver Cancer

02/06/2016 - 2 minutes

MiNA Therapeutics is launching the first saRNA human trial of its kind – OUTREACH for patients with severe liver cancer.

mina_therapeutics_biotech_rna_liver_cancer_trialMiNA 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.


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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.

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