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/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) |
| 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| B/BE/20/BVW6 | Dossier withdrawn by the notifier | Pfizer | Humans | Non-replicating recombinant vector derived from adeno-associated virus AAV6 serotype, containing a codon-optimised version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/18/BVW3 | A phase 2 study to assess the safety and efficacy of GM Lactococcus lactis for the attenuation of oral mucositis in subjects with cancers of the head and neck | Oragenics, Inc. | Humans | Biologically contained strain of Lactococcus lactis, genetically modified to secrete human Trefoil Factor 1 (hTFF1). |
| B/BE/18/BVW2 | A phase 2 vaccine study in healthy adults and adolescents to evaluate the safety and immunogenicity of two oral polio type 2 vaccine candidates | University of Antwerp | Humans | The nOPV2 candidate strains include different combinations of 5 distinct modified regions of the Sabin-2 genome, including changes to the RNA sequence in the 5’ untranslated region of polio genome (5’ UTR), the capsid protein coding region (P1), the non-structural protein 2C, and the polymerase 3D. Of these modifications, only the changes to polymerase 3D result in a change in the amino acid sequence. The rest of the modifications aim to stabilize the genetic sequence against reversion in either the 5’ UTR or capsid regions. |
| 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 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 |
| 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/11/V3 | Evaluation of the safety and efficacy of the vaccine PB-116 in the control of porcine pleuropneumonia caused by Actinobacillus pleuropneumoniae | Laboratorios Hipra S.A. | pigs | no new genes but deletion of a segment of gene A from which depends the haemolytic activity of the pathogen |