The Catalan biotech Gate2Brain, born from the University of Barcelona, IRB Barcelona and Hospital Sant Joan de Déu, has achieved a strategic milestone by receiving orphan drug designation from the United States Food and Drug Administration (FDA). This recognition, granted to its lead candidate G2B-002, intended for the treatment of aggressive pediatric brain tumors, has its roots in the research developed during the Industrial Doctorate project of Dr. Clàudia Resa, who completed her thesis on April 25, and who was interviewed by Pla DI.
The drug G2B-002 has been designed to combat forms of cancer such as diffuse intrinsic pontine glioma and pediatric glioblastoma, pathologies with very limited therapeutic options. The FDA grant, which is in addition to that already obtained by the European Medicines Agency (EMA) at the end of 2024, provides crucial regulatory support that will facilitate the transition from preclinical trials to clinical trials. “ This double recognition brings us one step closer to clinical trials and, ultimately, to offering new hope to young patients and their families ,” said Meritxell Teixidó, CEO and CSO of Gate2Brain, according to the news published by the Bosch i Gimpera Foundation.
This success of Gate2Brain cannot be understood without the strategic research carried out by Dr. Clàudia Resa within the framework of the Industrial Doctorate Plan of the Generalitat de Catalunya. Her project, a collaboration between the company and the research group in Pediatric Cancer Treatment of the Sant Joan de Déu Research Institute (IRSJD – UB), was decisive in generating the preclinical data that have supported the potential of G2B-002.
During her PhD, Dr. Resa focused on the preclinical evaluation of an innovative drug delivery system based on a shuttle peptide (MiniAp-4). This technology, patented by the company, allows highly effective chemotherapeutic drugs to cross the blood-brain barrier, one of the main obstacles in the treatment of brain tumors. “I am proud to have developed a new delivery system for a highly cytotoxic drug that until now was not administrable to patients,” explained Dr. Resa in a recent interview.
Her research demonstrated that this system not only transported the drug to the brain in relevant concentrations, but also achieved a significant reduction in tumor volume in animal models. These preclinical data, as the researcher herself confirmed, “facilitated the application for Orphan Drug Designation at the European level ” and have been the scientific basis for the recent designation by the FDA.
The case of Gate2Brain and Dr. Clàudia Resa exemplifies the value of the Industrial Doctorate model as an instrument to connect research talent with the needs of the productive fabric. Dr. Resa chose this modality due to her desire to “do applied research”, with the aim that the results would have a real impact beyond the academic field. The fluid collaboration between the research group and the company allowed the project to be oriented towards objectives with industrial viability and clinical relevance. “ Industrial doctoral students must always think about the applicability and industrial viability of their results, which gives them a broader perspective and one oriented towards real impact ”, reflected Dr. Resa in the interview conducted by Pla DI.
This synergy has culminated in international recognition that not only drives the future of Gate2Brain, but also validates the capacity of the Industrial Doctorate Plan to train highly qualified researchers and generate innovation with a direct return to society.
