Nanocarrier for small interfering ribonucleic acids to be used in oncology therapy

In the last years, high expectations were raised concerning the use of small interfering ribonucleic acids (siRNA) against gain-of-function gene products such as oncogenes in malignant neoplasia. However, therapeutic use of siRNAs was always compromised by their instability and missing cell specific carrier systems. Thus, the development of an efficient siRNA nano-carrier is a major goal to make use of RNA as a molecular therapeutic modality. In order to target siRNA efficiently, a German university applied the high RNA binding capacity of the small arginine-rich protein protamine for spontaneous electrostatic assembly of therapeutic nanoparticles decorated with tumor cell-specific antibodies. Fluorescence microscopy and dynamic light scattering measurements of these nano-carriers revealed the formation of a vesicular architecture. This architecture requires presence of antibody-protamine, defined excess of free SMCC (succinimidyl 4-(N-maleimidomethyl) cyclohexane-1-carboxylate)-protamine, and anionic siRNA to form. These complex nanoparticles were efficient in the treatment of non-small-cell lung cancer xenograft models, when the oncogene KRAS was targeted via epidermal growth factor receptor (EGFR)-mediated delivery. To show general applicability, the university used a modular platform. The university concludes that these antibody-protamine-siRNA nano-carriers provide a novel platform technology to specifically target different cell types and to date undruggable targets in cancer therapy by RNA interference (RNAi). Industrial partners from the field of oncology therapy, who would like to implement the solution in their treatments, are sought for licensing agreements that can be combined with research to further develop the solution.