Development of Patents and Clinical Trials on Regenerative Therapy: Gene Therapy

The most recent advances in biotechnology have transformed regenerative medicine from a mere promise to a therapeutic reality for a wide range of diseases. Gene therapy, understood as the introduction of nucleic acids within altered cells, is designed to improve or cure patients with a disease and is presented as an essential tool for the progress of this novel type of therapy. There are already controlled clinical trials on very different types of disease, applying new therapeutic approaches based on the utilization of genes to obtain stem cells. All of these investigations are generating spectacular developments and, in many cases, the inventions are being protected by patents due to the considerable interest of industry and their possible commercialization. Indeed, the development of different technologies aimed to generate induced pluripotent stem cells has been one of the most active areas in the patent production during the last year. We review the principles of gene therapy and describe in detail the different clinical trials and the diseases under investigation. We also outline the advances underlying the patents related to its application in regenerative medicine, the therapeutic interventions that have been carried out, and the limitations and shortcomings that remain to be overcome.