Clévio Nóbrega, principal investigator at the Algarve Biomedical Center Research Institute (ABC-RI) and professor at the Faculty of Medicine and Biomedical Sciences (FMCB) of the University of Algarve, has just received a project worth 1 million euros from the Viljem Julijan Association for Children with Rare Diseases, a Slovenian non-profit association, to develop a gene therapy for a rare disease.
The researcher traveled to the city of Ljubljana, Slovenia, this week to present the new project. On this occasion, he was received by the association’s founders, the scientific director, and members of the association. He also met with a set of companies and government entities.
This funding follows another one for the project “CureCSB – Development of a Gene Therapy for Cockayne Syndrome Type B,” funded with over 1 million euros, thus demonstrating the capacity of ABC-RI and the research laboratory led by Clévio Nóbrega to develop gene therapies for rare diseases in a preclinical context, at an international level.
According to Nejc Jelen, president of the Viljem Julijan Association for Children with Rare Diseases: “Very rare genetic diseases destroy the lives of affected children and their families. For this particular disease, with no cure or treatment and being fatal until now, there was no hope for the patients or their families. At this moment, we feel that there is hope, and in our association, we are very happy and excited to be able to fund Clévio Nóbrega’s research. We believe that this work is very important and will result in a gene therapy that will save the lives of children born with this disease and other rare diseases in the future.”
During this visit, Clévio Nóbrega also highlighted the ambitious nature of the project, emphasizing that “this new funding enables the creation of a platform for the development of gene therapies for rare diseases, which will be unique at the national level and innovative at the European level. It is a unique challenge for our laboratory, research center, and university, but with support and commitment, we will pave the way for true innovation and translation of research results to society.”
For this purpose, the research group already has the support of experts in both the clinical and pharmaceutical fields, ensuring close interaction with regulatory entities for the application of gene therapy in a clinical trial setting.
It should be noted that the Viljem Julijan Association for Children with Rare Diseases is primarily funded through fundraising activities, focusing on investment in relevant research and providing patients and their families with a glimpse of the future in terms of treatment or mitigation of the effects caused by these diseases.