ExPLoRNA developed a technology to modify a cap structure at 5’ mRNA end. This modification stabilizes mRNA and significantly increases its translation efficiency. The uniqueness of mRNA for vaccine development results from the fact that in a single inoculation multiple different mRNAs encoding various tumor-associated antigens can be administered to stimulate development of adaptive immune response. This is a huge advantage over recombinant vaccines that consist of single antigens. With stable and effectively translated mRNA multiple antigens can be directly delivered to professional antigen-presenting cells that induce protective immunity. Translation of mRNA leads to production of antigens that will stimulate both T-cells and B-cells, thereby mimicking development of natural immunity. The Project undertakes the verification of the safety of this vaccination immunotherapy and its ability to induce antigen-specific T-cells in the group of patients with malignant melanoma.
ExploRNA Therapeutics will achieve the aim of the Project by completing the following five phases of the research and development program:
(i) synthesis and analyzis of physicochemical and biological properties of cap analogs and modified mRNA;
(ii) synthesis optimization of the cap analogues on a larger scale and development of the mRNA synthesis methods;
(iii) pre-clinical studies in advanced cellular and animal models;
(iv) toxicology and pharmacokinetics in the GLP standard and selection of a candidate for clinical trials;
(v) phase I clinical trial in patients with malignant melanoma.
Project co-financed by the European Union from the European Regional Development Fund under the Smart Growth Operational Program.
Project implemented as part of the National Centre for Research and Development call: Fast Track POIR.01.01.01-00-0920/19
Total value of the project: PLN 25 995 670,81
Total value of funding: PLN 19 408 810,05