Search the database for deliberate release of GM medicinal products
Displaying 1 - 9 of 9
| EU record number | Title | Company / Sponsor | Treated organism | Genetic modification |
|---|---|---|---|---|
| B/BE/24/BVW5 | Phase 3, open-label, single-arm study to evaluate efficacy and safety of FIX gene transfer with PF-06838435 (rAAV-Spark100-hFIX-R338L) in adult male participants with moderately severe to severe hemophilia B (FIX:C ≤2%) (BeneGene-2) | Pfizer | Humans | Recombinant adeno associated viral vector containing the codon-optimised human factor IX (FIX) minigene (R338L variant) |
| B/BE/20/BVW6 | Dossier withdrawn by the notifier | Pfizer | Humans | Non-replicating recombinant vector derived from adeno-associated virus AAV6 serotype, containing a codon-optimised version of the human Factor VIII gene |
| B/BE/20/BVW4 | A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy | Pfizer | Humans | Non-replicating recombinant vector derived from adeno-associated virus AAV9, lacking all AAV viral genes and containing a miniaturized version of the human dystrophin gene |
| B/BE/19/BVW2 | A Phase 3 Open-Label, Single-Arm Study To Evaluate The Efficacy and Safety of BMN 270, an Adeno-Associated Virus Vector–Mediated Gene Transfer of Human Factor VIII in Hemophilia A Patients with Residual FVIII Levels Receiving Prophylactic FVIII | BioMarin Pharmaceutical Inc. | Humans | Recombinant adeno-associated virus serotype 5 (AAV5) containing a gene cassette expressing the human coagulation factor VIII (FVIII) gene |
| ADP-0055-001 | A phase I, ascending dose, to evaluate safety and efficacy of ADP A2M4CD8 in HLA-A2+ patients with MAGE-A4 psoitives tumors | Adaptimmune LLC | Humans | MAGE-A4 specific T cell receptor (TCR) with a CD8α co-receptor |
| EBOVEU001 | A phase IIa partial blind, placebo-controlled clinical trial to evaluate the safety and immunogenicity of the Ad5-EBOV in healthy adults aged between 18 and 60 years in Belgium. | Bejing Institute of Biotechnology, CanSino Biologics Inc, | Humans | Recombinant Adenoviral vector serotype 5 expressing the envelop glycoprotein from Ebola virus (Zaïre-Makona) |
| 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 | CRISPR Therapeutic AG | Humans | Modified cells expressing Hemoglobin F (HbF) |
| Only notified under the "contained use" procedure. Dossier submitted on 14/10/2015. | A phase II, single-arm, multi-center trial to determine the efficacy and safety of CTL019 in pediatric patients with relapsed and refractory B-cell acute lymphoblastic leukemia | Novartis Pharma Services AG | Humans | Chimeric antigen receptor against CD19 |
| B/BE/08/BVW1 | MI-CP178: Study of a Live, Attenuated Intranasal Vaccine Against Respiratory Syncytial Virus (RSV) and Parainfluenza Virus Type 3 (PIV3), in Healthy 6 to < 24 Month-Old Children and in 2 Month-Old Infants | MedImmune LLC | Humans | Genes of the human Parainfluenza virus fusion (F) and hemagglutinin-neuraminidase (HN) proteins; gene of the human Respiratory syncitial virus fusion protein (RSV F). |