Design and study of molecular transport systems to develop efficient photodynamic gene therapies against solid tumors (FOTOGEN)

Biomedical research is constantly searching for innovative tools to leverage advances in biotechnology to improve the study and treatment of diseases. In particular, the design of nucleic acids capable of performing specific functions within target cells is generating considerable interest due to its great potential in personalised medicine. These tools open the door to develop individualised treatments for various diseases, including cancer. However, the main challenge for applying these technologies is to ensure the safe and efficient delivery of nucleic acids into target cells. Furthermore, achieving effective treatments is not always possible, making it crucial to validate new combined therapies that broaden their scope of action.

Therefore, this project proposes designing novel molecular transport systems to develop efficient photodynamic gene therapies, based on the combination of cyclodextrins and porphyrins. Cyclodextrins are cyclic sugars able to bind to a wide variety of molecules, including nucleic acids. On the other hand, porphyrins have the ability to absorb light and trigger chemical changes in their environment. By combining these two molecules in a single system, we can significantly and controllably improve treatment characteristics such as bioavailability, pharmacokinetics and immune response, thanks to a technology called “photodynamic gene therapy.” This approach may allow to activate the therapeutic systems exactly where and when it is intended to work, but also to monitor its distribution in the organism in real time. Furthermore, maintaining a well-defined chemical structure ensures the reliability, reproducibility and consistency of these systems, crucial characteristics for their potential clinical application.