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 this week to the city of Ljubljana, in Slovenia, to present the new project, having, on this occasion, been received by the founders of the association, by the scientific director and members of the association, but also meeting with a number of companies and entities governmental.
This funding follows another one for the project “CureCSB – Development of a Gene Therapy for Cockayne Syndrome Type B”, funded by more than 1 million euros.
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 therefore mortal until now, there was no hope either for patients or their families».
«Right now we feel that there is hope and, in our association, we are very happy and excited to be able to fund the research of Clévio Nóbrega. We believe that this work is very important and that it will result in a gene therapy that will save the lives of children born with this disease and other rare diseases in the future", he added.
On the occasion of this visit, Clévio Nóbrega also highlighted the ambitious nature of the project.
“This new funding makes it possible to create a platform for the development of gene therapies for rare diseases, which will be unique at national level and innovative at European level. It is a unique challenge for our laboratory, research center and university, but with your support and commitment we will pave the way for true innovation and transfer of research results to society», he considered.
For this, the research group already has the support of specialists in both the clinical and pharmaceutical areas, guaranteeing, from the outset, a close interaction with regulatory authorities with a view to the application of gene therapy in the context of clinical trials.
The Viljem Julijan Association for Children with Rare Diseases is financed mainly through fundraising, investing 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.
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