Search the database for deliberate release of GM medicinal products
| EU record number |
Title |
Company / Sponsor |
Treated organism |
Genetic modification |
| B/BE/26/BVW3 |
A phase I/II/III study with a dose determination part followed by an efficacy and safety evaluation, quadruple blind placebo-controlled part and then by a long-term safety follow up part, in ambulant boys with Duchenne Muscular Dystrophy |
GENETHON |
Humans |
Non-replicating recombinant vector derived from adeno-associated virus AAV, lacking all AAV viral genes and carrying the human dystrophin (hMD1) gene |
| B/BE/26/BVW2 |
An Open-label, Multicenter, Two Part, Ascending Dose Followed by a Controlled Trial to Assess the Safety and Efficacy of a Subretinal Administration of AAVB-039 in Participants with Stargardt Disease (STGD1) (CELESTE) |
AAVantgarde Bio UK Ltd. |
Humans |
Non-replicating recombinant dual vector derived from adeno-associated virus AAV8 carrying functional copies of the ABCA4 gene. |
| Only notified under the "contained use" procedure. Dossier submitted on
. |
A multicentre, open-label, single ascending dose, dose-ranging, phase I/Iia study to evaluate the safety and tolerability of an autologous antigen-specific chimeric antigen receptor T regulatory cell therapy (TX200-TR101) in living donor renal transplant |
Sangamo therapeutics |
Humans |
chimeric antigen receptor specific to the donor HLA A*2 |
