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An international consortium, headed by the group of Prof. Dr. Frank Staal (Department of Immunohematology and Blood Transfusion, Leiden University Medical Centre) has been awarded a €6 million grant from the European Union to develop a gene therapy procedure for newborns suffering from severe combined immunodeficiency syndrome (SCID).

According to Prof. Dr. Frank Staal, the development of a gene therapy approach to cure SCID will provide a much needed treatment, because the standard treatment of stem cell transplantation is often not possible due to lack of a suitable donor. The approach to be developed under this Horizon2020 program will utilize a viral vector to deliver a healthy copy of the defective gene to the patients stem cells with the aim of correcting the clinical manifestations of SCID and dramatically increasing the life expectancy of SCID newborns. Under the program, Batavia Biosciences will employ its viral vector expertise and SIDUS® technology to develop and manufacture the clinical product in its GMP clean room facilities.

More information on website LUMC.