DNAlite Therapeutics is an early stage pharmaceutical platform developing nucleic acid medicines. mRNA has emerged as a leading therapeutic modality, but delivery remains to be a bottleneck. While current approaches inject mRNA into the bloodstream, DNAlite has created an oral approach to mRNA delivery that survives the stomach and gets taken up by epithelial cells in the gut. Once in the cells, the mRNA is read and the gut cells manufacture proteins — whatever is coded in the mRNA. These epithelial cells naturally turnover and refresh every 3 to 4 days, so DNAlite medicines could be taken on a regular cadence. The proteins expressed can be antibodies and other biologics; it can be equivalent to rare disease gene therapy, but essentially reversible, because no permanent changes are made.

The company has demonstrated proof of platform in-vivo, producing and quantifying proteins from orally given mRNA creating a completely new modality with broad and unique value. They are slated to enter the clinic with IL-22 as their first therapeutic target to tackle indications such as Inflammatory bowel disease (3 Mn
patients in US), Acute graft versus host disease of the gut (incidence of ~5000/yr), and Necrotizing enterocolitis (incidence: ~400/yr in the US).