| B/BE/18/BVW6 | Global study of a recombinant adeno-associated virus designed to address the monogenic root cause of Spinal Muscular Atrophy | Humans | Recombinant adeno-associated virus serotype 9 (AAV9) containing the cDNA of the human SMN (Survival Motor Neuron) gene | 
          
                  | 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 09/11/2001. | A phase III, multi-center, open-label, randomized study to compare the overall survival and safety of bi-weekly intratumoral administration of INGN 201 versus weekly methotrewate in 240 patients with refractory squamous cell carcinoma of the head and neck | Humans | Wild-type p53 | 
          
                  | Only notified under the "contained use" procedure. Dossier submitted on 09/11/2001. | A phase III, multi-center, open-label, randomized study to compare the effectiveness and safety of intratumoral administration of INGN 201 in combination with chemotherapy versus chemotherapy alone in 288 patients with recurrent squamous cell carcinoma of | Humans | Wild-type p53 |