Development of Innovative Therapeutic Solutions Using RNA Technology

Co-financing from the state budget from the Medical Research Agency

Competition: Development of Innovative Therapeutic Solutions Using RNA Technology

The goal of the "TransformRNA" Project is to develop clinical candidates based on mRNA technology in 3 therapeutic areas (oncological, metabolic - AATD deficiency and viral - SARS-CoV2), as well as to develop and expand the competence of Celon Pharma S.A. in the area of pharmaceutical production in the area of mRNA - launching and qualifying a sterile production zone in the GMP standard, capable of supplying the domestic market with vaccines in case of pandemic-related needs. The overriding goals of the Project are to develop and provide the society with new generation therapies, both in the field of antiviral vaccines, in metabolic and oncological diseases, and the development of mRNA antibodies. Achieving these goals will bring significant benefits to society by improving treatment efficiency for these severe and difficult-to-treat diseases.

The "TransformRNA" project includes, in the first stage, basic research, as a result of which therapeutic goals will be confirmed and validated. In the next stages of preclinical work, drugs based on mRNA technology will be developed, the safety and effectiveness of which will then be verified in the first and second clinical phase. In addition, the Project will ensure the improvement and optimization of the existing production infrastructure, and selected investments (including the purchase of selected GMP of fill and finish production line equipment) will allow for the production of mRNA products on a scale sufficient for key clinical trials, as well as for market needs. In summary, the project has the following goals, clinical evaluation as part of the planned clinical phases I and II of at least three drug candidates, including one mRNA vaccine against SARS-COV-2. The project also aims to expand Celon Pharma's competence in the design and optimization of mRNA therapeutics and their production in the GMP standard, formulations in LNP technology and release to the market.

Co-financing value: PLN 83,556,811.52

The total cost of the project: PLN 141,630,945.59