710195Development of siRNA Nano-Carriers for In Vivo R&D and Gene TherapyClosedGRD Proof of ConceptInnovate UKNucleic acids are at the very center of all cellular processes, with chromosomal DNA serving as a storage device for genetic information and RNA molecules directly controlling gene expression and, through this, each and every cellular function. Consequently, the clinical application of short RNA molecules (e.g. miRNA, siRNA) to “silence” the expression of specific malignant genes (process known as RNA interference) can be used to treat both genetic and acquired diseases (e.g. cancers and viral infections) at their origin, not merely their symptoms. Stimulated by its therapeutic potential and outstanding advances in preclinical gene therapy and regenerative medicine, the use of RNA interference (RNAi) in R&D and Pharma is a fast growing sector of the biomedical market. There is, however, a major technical hurdle: the transport of nucleic acids through cell membranes is blocked by their size and negative charge. To facilitate the intracellular delivery of siRNA, DELIVERICS developed a ground-breaking technology based on biodegradable nano-carriers able to carryout this challenging task at very high efficiency without toxicity; a technology currently commercialised as R&D kits for “in vitro” studies: SAFEctin Transfection Reagent and SAFEctin-STEM. Aiming to develop a gene therapy drug able to treat skin lesions by topical administration, DELIVERICS (in collaboration with the University of Edinburgh) aims to optimise its platform technology for siRNA delivery into skin cells. This biodegradable vehicle, so-called LIFEctin LOCAL, will address a commercial opportunity that exists in both the R&D and Pharma sectors, where siRNA delivery tools with clinical-grade safety and reliability are required for "in vivo" R&D studies and for developing efficient RNAi-based drugs.