GuidePro

A recently Nobel-prize winning technology, CRISPR, is making its way into the clinic, offering promise in enhancing the health and life expectancy of people affected by diseases that lack any existing treatment. The technique is also called molecular scissor, as it allows for cutting/correcting a diseased gene at a very precise position. For this application to work, a so-called guide RNA is needed to bring the scissor to the desired position in the gene. This guide RNA is designed specifically for each application/position in a gene to be cut or corrected. Once designed, it has to be produced with exactly the required sequence and with sufficient purity to avoid unwanted cuts somewhere else in the genes. It also needs to be stabilised to avoid a quick degradation in body fluids. The project addresses the challenges related to the chemical production of gram quantities of these guide RNAs, their purification and quality control in order to reach a product quality suitable for human application. In fact, the synthesis of guide RNA represents about 400 chemical reactions versus roughly 80 for more standard RNA medicines. The length of these molecules as well as their structural characteristics makes the purification also very difficult and heavily complicates the quality control methods to ensure suitability for human use. The final goal of the work package is therefore to develop a robust and scalable production methodology for guide RNAs suitable for use in human therapeutic applications.

The necessary quality control (QC) methods for ensuring purity, safety and necessary characterisation will be developed by our partner Quality Assistance, a leading Contract Research Organisation (CRO) providing the pharmaceutical industry with all the analytical services required by EMA and FDA regulations for the development and marketing of innovative human medicinal products.