cmRNAbone

Due to demographic change, bone degenerating diseases like osteoporosis have a growing societal and financial impact on industrialised nations. It is estimated that worldwide one in three women and one in five men over the age of 50 will experience osteoporotic bone fracture. Moreover, although most fractures will heal without complications under appropriate treatment, there are additional cases of impaired bone regeneration, e.g. large trauma with infections after car accidents.

With bone being the most transplanted tissue after blood, the need for graft materials is enormous. In search of optimised regeneration solutions, the EU-funded cmRNAbone project has set out to develop a novel gene therapy to improve the lives of people with large traumatic injuries or bone degenerating diseases such as osteoporosis.

The proposed approach is a unique combination of genetic research, advanced nano- and biotechnology and 3D-printing: Making use of recent scientific discoveries related to RNA therapeutic agents, the consortium aims to develop chemically modified RNA encoding specific proteins targeting neurogenesis, vasculogenesis and osteogenesis – three major processes influencing the healing progression. The produced RNA sets will be combined with non-viral vectors for the RNA delivery to be embedded in a biomaterial ink formulation. The use of a specifically designed 3D-printer for the implant will help to demonstrate the bone regenerative capabilities in practice.

The new findings will be applied in two parallel pre-clinical studies to demonstrate clinical relevance and validity of the designed therapeutic in osteoporotic and critical size bone defects. Supported by a clinical and scientific advisory board, the largely SME-driven consortium will insure facile and rapid translation to the clinic beyond the end of the project. In the long run, the discoveries could not only be a new regenerative approach in large bone defect and fragile fractures in the young and ageing population, but also to other major diseases affecting millions of patients.

The cmRNAbone consortium comprises 11 European partners from Austria, France, Germany, the Netherlands, Spain and Switzerland.

CIDETEC Nanomedicine´s main role in the project is to develop optimized non-viral polysaccharide-based cmRNA delivery vehicles suitable for the matrix developed targeting guidance of tissue regeneration.