Search the database for deliberate release of GM medicinal products

Displaying 1 - 11 of 11
EU record number Title Company / Sponsor Treated organism Genetic modification
B/BE/21/BVW8 Phase 3 Randomized, Controlled Study of AAV5-hRKp.RPGR for the Treatment of X-linked Retinitis Pigmentosa Associated with Variants in the RPGR gene Meira GTx UK II Limited Humans Non-replicating recombinant vector derived from adeno-associated virus AAV 5 carrying the human RPGR gene
B/BE/20/BVW6 Dossier withdrawn by the notifier Pfizer Humans Non-replicating recombinant vector derived from adeno-associated virus AAV6 serotype, containing a codon-optimised version of the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
CYAD-211-001 An open-label phase I, multi-center study to determine the recommended dose of the chimeric antigen receptor T-cell treatment CYAD-211 after a non-myeloablative preconditioning chemotherapy in multiple myeloma patients with relapsed or refractory disease. Celyad Humans BCMA-CAR and ShRNA CD3ζ
CYAD-N2T-005 An open-label, Phase I/II study to assess the safety and clinical activity of NKR-2 treatment administration after non-myeloablative preconditioning chemotherapy in relapse/refractory acute myeloid leukemia or myelodysplasic syndrome patient (DEPLETHINK - Celyad Humans NKG2D-chimeric antigen receptor
CYAD-02-001 An open-label, phase I, multi-center study to determine in relapsed/refractory acute myeloid leukemia or myelodysplasic syndrome patients the recommended dose of CYAD-02 after a non-myeloablative preconditioning chemotherapy followed by a potential CYAD-0 Celyad Humans NKG2D-chimeric antigen receptor, ShRNA targeting the endogenous RNA of NKG2D ligands MICA and MICB and truncated CD19 reporter protein
CYAD-N2L-101 An open-label, Phase I study to assess the safety of multiple doses of CYAD-101, administered after standard FOLFOX chemotherapy in patients with unresectable metastatic colorectal cancer (alloSHRINK-study-Standard cHemotherapy Regimen and Immunotherapy w Celyad Humans NKG2D-chimeric antigen receptor, the truncated CD19 tag and the TIM8 molecule which interferes with the interaction between the natural TCR and endogenous CD3ζ
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)
CYAD-N2T-006 An open-label, phase I study to assess the safety of NKR-2 treatment administrated concurrently with 5-azacytidine in tretment-naïve acute myeloid leukemia or myelodysplastic syndrome patients not candidates for intensive therapy (EPITHINK - EPIgenetic dr Celyad Humans NKG2D-chimeric antigen receptor
Only notified under the "contained use" procedure. Dossier submitted on 25/10/2016. A multinational, open-label, dose escalation Phase I/II study to assess the safety and clinical activity of multiple administrations of NKR-2 in patients with different metastatic tumor types Celyad Humans NKG2D-chimeric antigen receptor
Only notified under the "contained use" procedure. Dossier submitted on 08/07/2010. An open-label, phase I, dose-escalation and safety study of two intramuscular injections at dose of 2.9 log or 4 log CCID50 of the recombinant HIV I clade B measles vaccine vector in healthy adults. Institut Pasteur - Paris Humans Gag, Pol, Nef genes of HIV