Inhibiting certain enzymes in the cells of the intestine could be beneficial to patients suffering from Crohn's disease. One of the potential treatments being addressed by the EU-backed NewDeal project would use RNAi (or RNA interference) to locally inhibit the synthesis of the concerned proteins (JAK1 and JAK3 of the Janus kinase family of enzymes) in a targeted and transitory manner. Specifically, short interfering RNA (siRNA) would be used to silence the expression of the proteins' genes.

IDIBAPS, a biomedical center in Barcelona, Barcelona University Medical Center , also a partner on the project, helped to identify the biological targets. BIG will develop the siRNA. Leti developed the RNA vector: lipid nanoparticles capable of crossing both the intestinal barrier and cell membranes to effectively carry the siRNA therapeutics to the intestinal cells being targeted. The drug and vector are contained in a special polymer capsule, a system designed by a pharmaceutical company to protect their contents along the GI tract after oral administration.. The objective of the project–still in its early days–is to validate all stages up to Phase 1 clinical trials within four years.

*Bioscience and Biotechnology Institute of Grenoble