| 
            Only notified under the "contained use" procedure. Dossier submitted on 20/08/2018.           | 
                  
            A Phase I/II study of the safety and efficacy of a single dose of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells in subjects with severe sickle disease           | 
                  
                  
            Humans           | 
                  
            Modified cells expressing Hemoglobin F (HbF)           | 
              
          
                  | 
            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)           | 
              
          
                  | 
            Only notified under the "contained use" procedure. Dossier submitted on 24/04/2017.           | 
                  
            A Phase 1, Open Label, Non-comparative Study to Evaluate the Safety and the Ability of UCART19 to Induce Molecular Remission in Paediatric Patients With Relapsed/Refractory B Acute Lymphoblastic Leukaemia           | 
                  
                  
            Humans           | 
                  
            chimeric antigen receptor and suicide-ligand (RQR8)           |