Description
Objectives
The main objective of the group is the development of advanced therapy drugs covering all phases, from basic research and preclinical studies to the evaluation of the efficacy and safety of these drugs in humans (clinical trials).
Some of these advanced therapies are already being manufactured for clinical use in our IBGM Cell Production Unit (UPC-IBGM), created in 2007 as the first clean room promoted in Spain by the public sector to support clinical trials in the National Health System.
In collaboration with clinical groups from different institutions (IOBA, Hospital Río Hortega, Hospital Clínico Universitario de Valladolid, Hospital General de Oviedo, and ITRT of the Clínica Quirón-Teknon in Barcelona), several clinical trials have been carried out, authorized by the AEMPS and financed by regional and national governments, to test the efficacy and safety of the advanced therapy drugs developed by the group in the treatment of various pathologies: cardiac (myocardial infarction), ophthalmological (repair of damaged ocular surface), osteoarticular diseases (lumbar degenerative disc disease, knee osteoarthritis, tendinopathy), autoimmune disorders (lupus), with very promising results that have led to the publication of numerous scientific articles.In addition, the group is researching on the improvement of mesenchymal cell culture processes for clinical use and the optimization of viable alternatives (donor cells for allogeneic use and cryopreserved cells, among others) that reduce logistic and production costs and that, in the near future, will make cell therapy treatments possible in a standardized manner within the health system.
Another of the group's priority objectives is the development of new advanced therapy drugs. We have several projects underway aimed at developing innovative drugs that include the latest genetic engineering techniques based on CRISPR-Cas9. One of them involves the production of T lymphocytes genetically modified to express a specific receptor that directs their cytotoxic action against tumor cells (CAR-T cells).The aim is to develop this therapy for allogeneic use, from donor cells, by including the necessary genetic modifications and optimizing the culture conditions. On the other hand, we are also investigating CRISPR-Cas9-based strategies to combat noise-acquired hearing loss in humans.This project combines knowledge on the molecular basis of sensory differentiation with genetics and cell and gene therapy.The results will generate fundamental knowledge and protocols for the replacement or regeneration of defective cells through gene correction of patient cells in vivo and thus pave the way for a therapy to cure deafness.
Research lines
- Development and optimization of cell therapy drugs based on bone marrow mesenchymal cells (MSV®, authorized by the AEMPS for clinical use).
- Study of the efficacy and safety of MSV® in degenerative pathologies (osteoarticular, ophthalmological) or related to the immune system (lupus, COVID-19).
- New model of cell therapy against cancer based on CAR-T cells for allogeneic use.
- CRISPR-Cas9-based therapies against noise-acquired hearing damage.