Search the database for deliberate release of GM medicinal products
Displaying 1 - 10 of 10
EU record number | Title | Company / Sponsor | Treated organism | Genetic modification |
---|---|---|---|---|
B/BE/24/BVW4 | A phase 3 multinational, open-label, systemic gene delivery study to evaluate the safety and efficacy of SRP-9003 in subjects with limb girdle muscular dystrophy 2E/R4 | Sarepta Therapeutics | Humans | Non-replicating recombinant vector derived from adeno-associated virus AAV, serotype rh74, lacking all AAV viral genes and carrying the full-length sarcoglycan-beta (SGCB) gene |
B/BE/22/BVW6 | A phase 3, Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of SRP-9001 in Non-Ambulatory and Ambulatory Subjects With Duchenne Muscular Dystrophy (ENVISION) | Sarepta Therapeutics | 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 |
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) | F. Hoffmann-La Roche | 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 |
B/BE/22/BVW4 | A Phase I/II, Multicenter, Open-Label Study of Nous-209 Genetic Vaccine for the Treatment of Microsatellite Unstable Solid Tumors | Nouscom Srl | Humans | The study involves two GMOs: (i) A replication-incompetent adenovirus (GAd20 with deletions of the viral E1, E3 and E4 coding regions) isolated from a gorilla and encoding the FSP neoantigens; (ii) an attenuated, replication-defective orthopoxvirus (Modified Vaccinia virus Ankara), encoding for the same neoantigens. |
B/BE/21/BVW5 | A Phase 3 Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Delivery Study to Evaluate the Safety and Efficacy of SRP-9001 in Subjects With Duchenne Muscular Dystrophy (EMBARK) | Sarepta Therapeutics | 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 |
B/BE/21/BVW3 | Phase I/II study to evaluate the safety and effects on Progranulin levels of PR006 in patients with Fronto-Temporal Dementia with Progranulin mutations (FTD-GRN). | Prevail Therapeutics | Humans | The study involves a replication-incompetent adeno-associated virus, AAV9, lacking all AAV viral genes and encoding for the human Progranulin (GRN) gene |
Only notified under the "contained use" procedure. Dossier submitted on 03/05/2021. | Administration of genetically engineered T cells (KTE-X19) in Pediatric and Adolescent Subjects with Relapsed/Refractory B-precursor Acute Lymphoblastic Leukemia or Relapsed/Refractory B-cell Non-Hodgkin Lymphoma | Kite Pharma | Humans | CD19-CAR |
B/BE/20/BVW5 | Phase 1b Study to Evaluate the Safety, Tolerability, and Anti-Tumor Activity of Nous-PEV, with pembrolizumab, in Patients with Unresectable Stage III / IV Cutaneous Melanoma and with Stage IV NSCLC | Nouscom Srl | Humans | The study involves two GMOs: (i) A replication-incompetent adenovirus (GAd20 with deletions of the viral E1, E3 and E4 coding regions) isolated from a gorilla and encoding the PEV neoantigens gene; (ii) an attenuated, replication-defective orthopoxvirus (Modified Vaccinia virus Ankara), encoding for the neoantigens gene. |
COVID-19-101 | A randomized, placebo-controlled trial, to evaluate the safety and immunogenicity of the COVID-19 vaccine, a measles vector-based vaccine candidate against COVID-19 in healthy volunteers consisting of an unblinded dose escalation and a blinded treatment p | Institut Pasteur & Themis Bioscience GmbH | Humans | Recombinant measles virus vaccine strain (Schwarz strain) expressing the Spike glycoprotein 1 from SARS-CoV2 |
Only notified under the "contained use" procedure. Dossier submitted on 25/04/2018. | A Phase 3, Randomized, Open-Label Study Evaluating the Efficacy of Axicabtagene Ciloleucel versus Standard of Care Therapy in Subjects with Relapsed/Refractory Diffuse Large B Cell Lymphoma (ZUMA-7) | Kite Pharma | Humans | Chimeric antigen receptor against CD19 |