Certain miRNAs are central regulators in cancer, acting in complex networks as either oncogenes or tumor suppresors. MiRNAs silence specific target messenger RNAs (mRNAs) through complementary binding. They act as central regulators of disease and can target multiple RNAs simultaneously. This means that a single miRNA can have a very powerful and deep effect on a cell’s behavior.
miRNAs and Cancer
Mature miRNAs serve many functions in the cell, where they act through a process called gene silencing.
In cancer, tumor suppressing miRNAs are downregulated, leading to accumulation of target mRNAs with oncogenic potential.
Our focus is on the latter: when tumor suppressing miRNA molecules become dysregulated in a cancer, tumor cells can become more aggressive and resistant to other therapies. Our goal is to replace the missing miRNAs with specially designed substitute mimics, so called because they imitate the natural effects of the miRNAs that have gone missing.
MiRNAs are considered one of the most promising new types of drugs; their small size, known and conserved sequence, and central targeting focus, as well as their relative ease of production, make them attractive candidates from a drug development standpoint.
At Speratum, we are developing technologies that can harness the power of tumor suppressing microRNAs by restoring their levels in cancers.
Our lead therapeutic candidate, miR-198, is a miRNA replacement therapy for pancreatic cancer, a particularly aggressive cancer type, with current 5-year survival estimates in the single digits–and resistant to our current therapies. We believe our therapeutic platform has the potential to change that.