Sickle Cell Disease (SCD) and Beta Thalassemia are severe blood disorders that together affect more than 5 million people around the world. Both are inherited diseases caused by mutations in the HBB gene, which is responsible for producing a key component of hemoglobin. Without this component, red blood cells become misshapen and are less efficient at transporting oxygen around the body.

Patients with these diseases experience painful symptoms, require chronic hospitalisation and face early mortality. Currently, patients have very limited access to curative treatments that are also highly invasive and entail severe, life-long, risks.

The CEBPA gene encodes CCAAT/enhancer binding protein alpha (C/EBP-α), a transcription factor that acts as a master regulator of cell lineage determination and differentiation of myeloid cells and other cells types around the body. Myeloid cells are frequently dysregulated in the microenvironment of solid tumours and are understood to be an important resistance hurdle for many anti-cancer medicines. Restoring CEBPA expression in myeloid cells is hypothesised to alter immune cell populations in the tumour microenvironment and improve the efficacy of cancer therapies in solid tumour malignancies.