ABC-RI researcher receives project of more than one million euros to develop therapy for rare and incurable disease

Clévio Nóbrega, principal investigator at the Algarve Biomedical Center Research Institute (ABC-RI) and professor at the Faculty of Medicine and Biomedical Sciences at the University of Algarve, has received a project of more than one million euros to develop a gene therapy for the Cockayne syndrome disease, a rare and incurable disease that mainly affects children, with a prevalence of about 2.7 per million births in Western Europe.

As there is, so far, no therapy that modifies the progression of the disease, the UAlg researcher’s project aims to find in the genes a response that, if successful, could modify the progression of the disease and not just its symptoms.

Clévio Nóbrega and his research team aims, through the project “CureCSB – Development of a Gene Therapy for Cockayne Syndrome Type B”, to develop, over the next two years, a gene therapy for Cockayne Syndrome, type B, which reveals the potential to reach the stage of clinical trials and can reach the clinical area in the near future, changing the lives of patients and their families.

In the words of Clévio Nóbrega, principal investigator, “the path is long”, but the team leaves a guarantee: “we are working very focused so our wish is that the path can be covered in the shortest possible time and with the best results.”

For this purpose, the research group already has the support of specialists in both, the clinical and pharmaceutical areas, ensuring a close interaction with the regulatory authorities, in order to a future application of gene therapy in the context of clinical trials.

Developed within the Algarve Biomedical Center (ABC), this project is, for Nuno Marques, President of ABC, proof that “ABC-RI has the appropriate technology, team and recognition to contribute in a very fruitful way to the developments in medicine internationally”. The president guarantees that “the commitment to development and innovation is a winning bet, including regional and national economic diversification”.

Isabel Palmeirim, director of the Faculty of Medicine and Biomedical Sciences, shares the same conviction. She believes that this project is very important because “it reflects the vision of an academy increasingly focused on innovation, discovery and in dialogue with society problems”. As he states, “the University of Algarve and the Faculty of Medicine and Biomedical Sciences will continue to develop their work in order to strengthen the country and the region, providing them with new skills in the health sector”.

This new funding opens up new perspectives to understand a poorly studied disease that could bring, in the near future, a new way of looking at the disease.