Search the database for deliberate release of GM medicinal products
Displaying 1 - 4 of 4
EU record number | Title | Company / Sponsor | Treated organism | Genetic modification |
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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/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 |
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 24/04/2017. | A Phase 1, Open Label, Non-comparative Study to Evaluate the Safety and the Ability of UCART19 to Induce Molecular Remission in Paediatric Patients With Relapsed/Refractory B Acute Lymphoblastic Leukaemia | Institut de Recherches Internationales Servier | Humans | chimeric antigen receptor and suicide-ligand (RQR8) |