Professor Amit Nathwani, a NIHR Senior Investigator, is the Director of the Katharine Dormandy Haemophilia Centre at the Royal Free Hospital and Professor of Haematology at UCL. He is internationally recognised for his pioneering work in gene therapy and is the recipient of the ESGCT Outstanding Achievement Award and the Human Gene Therapy Award amongst others. Prof Nathwani developed a novel gene transfer approach that enabled the first successful trial of gene therapy in a bleeding disorder- haemophilia B and this has now become the platform for a variety of genetic disorders. He has also developed a novel approach for gene therapy of haemophilia A that has resulted in normalisation of coagulation factor VIII levels and correction of bleeding diathesis in severely affected patients. His group is working on other translational gene transfer approaches for a range of monogenetic disorders as well as malignancies.