Through transcriptional activation, saRNA therapeutics promise a revolution in our ability to modulate previously undruggable targets.
Through transcriptional activation, saRNA therapeutics promise a revolution in our ability to modulate previously undruggable targets.
DEVELOPMENT PROGRAMS
MTL-CEBPA
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.
For more information on MTL-CEBPA check out these publications:
MTL-CEBPA is a new medicine initially being developed as a combination therapy in cancer. MTL-CEBPA is designed to reduce immune suppression of myeloid cells by restoring C/EBP-α protein to normal levels using the RNA Activation mechanism. MTL-CEBPA has been tested in a range of pre-clinical cancer models and has been shown to enhance the anti-tumour activity of a standard of care cancer treatments.
MTL-CEBPA has been evaluated in clinical trials in more than 120 subjects. MTL-CEBPA has been evaluated in patients with advanced liver cancer; as a monotherapy in 39 patients and in combination with standard of care Sorafenib in 36 patients. In addition MTL-CEBPA has been evaluated in 50 patients with advanced solid tumor malignancies in combination with cancer immunotherapy Pembrolizumab.