Ascidian Therapeutics is creating gene therapies that replace exons via selective splicing of pre-mRNA. The technology is called RNA exon editing. A designer RNA is used to replace mutated, disease-causing exons within pre-mRNA. Multiple continuous exons can be replaced. This gene therapy approach allows editing large portions of RNA (rather than just single base changes), avoids the risks of direct DNA editing, and does not alter endogenous gene expression levels.
The company is named Ascidian, because their technology is based on a biological process that naturally occurs in ocean creatures called ascidians (also known as sea squirts). Ascidians use unique mechanisms of RNA splicing to re-engineer their transcriptome in developing from larvae to adults.