In all of us, RNA is the messenger between DNA and proteins. Recent discoveries in molecular biology have revealed that RNA is also involved in the regulation of genome organization and gene expression. These regulatory RNAs – or regRNAs – play a crucial role in controlling gene expression. By precisely targeting regRNA, we can increase gene expression in a specific and tunable way.
Upregulating gene expression with intentionally-designed drugs that target regRNA opens up a vast frontier of opportunity to develop truly transformative medicines. Our therapeutic approach is applicable to hundreds of diseases caused by genetic anomalies that can be addressed by increasing the output of a certain gene.
A genetic mutation damages one gene copy, eliminating protein expression
Leading to ~50% of normal protein levels.
Recessive or Loss-of-Function Diseases
Genetic mutations damage both gene copies, reducing protein production
Leading to <25% of normal protein levels.
Our initial focus is on diseases of the liver and central nervous system. We believe our platform is applicable to every tissue in the human body and we see vast potential.