| 
            B/BE/22/BVW5           | 
                  
            A two-part, open-label systemic gene delivery study to evaluate the safety and expression of RO7494222 (SRP-9001) in subjects under the age of four with Duchennne Muscular Dystrophy (ENVOL)            | 
                  
                  
            Humans           | 
                  
            Non-replicating recombinant vector derived from adeno-associated virus AAV, serotype rh74, lacking all AAV viral genes and carrying the human micro-dystrophin (hMicro-Dys) gene           | 
              
          
                  | 
            Only notified under the "contained use" procedure. Dossier submitted on 02/12/2019.           | 
                  
            A multicentre, open-label, single ascending dose, dose-ranging, phase I/Iia study to evaluate the safety and tolerability of an autologous antigen-specific chimeric antigen receptor T regulatory cell therapy (TX200-TR101) in living donor renal transplant            | 
                  
                  
            Humans           | 
                  
            chimeric antigen receptor specific to the donor HLA A*2           | 
              
          
                  | 
            Only notified under the "contained use" procedure. Dossier submitted on 21/02/2018.           | 
                  
            Efficacy and safety of bilateral intravitreal injection of GS010: a randomized, double-masked, placebo-controlled trial in subjects affected with G11778A ND4 leber hereditary optic neuropathy for up to one year.           | 
                  
                  
            Humans           | 
                  
            human wild-type MT-NDA (mitochondrial NADH Dehydrogenase 4)           |