His work combined scientific vision, experimental rigor and a constant focus on the potential impact of research for patients affected by severe inherited diseases.
Fondazione Telethon mourns the passing of Professor Claudio Bordignon, a pioneer of gene therapy and the first Director of the San Raffaele-Telethon Institute for Gene Therapy.
Professor Bordignon was among the scientists who, at a very early stage in the history of gene therapy, helped transform a bold scientific idea into a concrete therapeutic strategy. His work combined scientific vision, experimental rigor and a constant focus on the potential impact of research for patients affected by severe inherited diseases.
Among his most significant contributions was his pioneering role in the development of gene therapy for adenosine deaminase-deficient severe combined immunodeficiency (ADA-SCID), one of the disorders that most clearly demonstrated the therapeutic promise of gene transfer.
His early studies show that retroviral vectors could be used to introduce a functional ADA gene into hematopoietic cells, providing an essential proof of principle for the field and leading to the approval of the first gene therapy clinical trials in the US and Italy for a genetic disease. The early clinical studies in ADA-deficient patients showed that genetically modified peripheral blood lymphocytes and bone marrow cells could engraft, express the therapeutic gene and produce measurable biological benefit.
These achievements represented crucial early milestones for gene therapy, demonstrating that gene therapy could move beyond theory and become a real path toward treatment. These early studies established the scientific and clinical foundations for the subsequent advances that led to the first successful treatment of ADA-SCID with hematopoietic stem cell gene therapy by his team.
As first Director of the San Raffaele-Telethon Institute for Gene Therapy, Professor Bordignon also played a decisive role in creating an environment in which cutting-edge research could be translated into clinical development. His leadership helped lay the groundwork for the results that, in the years that followed, would bring concrete benefits to patients with rare genetic diseases. He helped establish Italy’s first private GMP facility for the manufacture of advanced therapy medicinal products in the San Raffaele biocluster, guided by his early vision that only high-quality manufacturing and controlled processes could turn pioneering science into therapies for patients.
Beyond inherited diseases, he helped extend gene therapy into oncology and immunotherapy, contributing to early work on genetically modified donor T cells designed to enhance graft-versus-tumor activity while controlling complications such as graft-versus-host disease. As a founding member of the European Research Council, first President of European Society of Cell and Gene Therapy and member of several Italian government’s scientific committee he played an important role in fostering the development of young scientists, supporting the emergence of a new generation of researchers in Italy and across Europe.
Fondazione Telethon expresses its deep gratitude for Professor Bordignon’s scientific vision, his pioneering contribution to the field of gene therapy, and his role in opening a path that would later change the outlook for many patients and families. We remember him with respect, gratitude and admiration, and we extend our sincere condolences to his family, colleagues and all those who worked with him over the years.
