A gene therapy based clinical trial for Fanconi anemia patients offers its first successful results

The prestigious journal Nature Medicine publishes the first results of a gene therapy based clinical trial in patients with Fanconi anemia. In this study the authors demonstrate for the first time the production of blood cells derived from the patients’ genetically corrected stem cells.
This clinical trial has been carried out by members of a novel research program, EUROFANCOLEN, coordinated by Dr Juan Bueren, from the Center for Energy, Environment and Technology (CIEMAT), the Center for Biomedical Network Research on Rare Diseases (CIBERER) and the Institute of Health Research of the Jiménez Díaz Foundation (IIS-FJD), under the clinical direction of Dr Julián Sevilla, from the Foundation of the Hospital del Niño Jesús, in Madrid, the clinical sponsor of the trial. The first authors of the study are Dr. Paula Río and Dr. Susana Navarro, affiliated to the CIEMAT / CIBERER / IIS-FJD Consortium. The gene therapy vector was developed in collaboration with Genethon and produced at YposKesi in France.