Search the database for deliberate release of GM medicinal products
Displaying 1 - 5 of 5
| EU record number | Title | Company / Sponsor | Treated organism | Genetic modification |
|---|---|---|---|---|
| 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 |
| B/BE/18/BVW7 | Phase III trial investigating a AAV vector containing a variant of human factor IX gene administered to adult subjects with severe or moderately-severe hemophilia B | uniQure biopharma B.V. | Humans | Recombinant adeno associated viral vector containing the wild type modified to express the Padua derivative of human coagulation factor IX cDNA |
| B/BE/18/BVW5 | A Phase 1b/2a study to assess the safety and tolerability of GM Lactococcus lactis in subjects with Type 1 Diabetes Mellitus | Intrexon T1D Partners, LLC | Humans | Biologically contained strain of Lactococcus lactis, genetically modified to secrete human interleukin-10 and proinsulin |
| 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 04/05/2018. | Phase IIa clinical trial for the evaluation of a vaccin against influenza virus H3N2 | FluGen Inc. | Humans | recombinant H3N2 influenza virus: 6 intern genes of the replication defective A/Puerto Rico/8/34 (PR8) strain with hemagglutinine and neuraminidase from A/Brismane/10/2007 (H3N2) strain |