Search the database for deliberate release of GM medicinal products
EU record number
Title
Company / Sponsor
Treated organism
Genetic modification
B/BE/25/BVW8
A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Investigate the Efficacy of a Single Intravenous Dose of SGT-003 in Ambulant Males With Duchenne Muscular Dystrophy Solid Biosciences Inc.
Humans
Non-replicating recombinant vector derived from adeno-associated virus AAV, lacking all AAV viral genes and carrying the human micro-dystrophin (h-µD5) gene
Only notified under the "contained use" procedure. Dossier submitted on 2 September, 2022
.
A single-arm, open-label, multi-centre, phase I/II study evaluating the safety and clinical activity of QEL-001, an autologous CAR T regulatory cell treatment targeting HLA-A2, in HLA-A2/ A28neg patients that have received an HLA-A2pos liver transplant Quell Therapeutics Limited
Humans
BCMA CAR; shRNA CD3ζ; tCD34
Only notified under the "contained use" procedure. Dossier submitted on 21 February, 2018
.
Efficacy and safety of bilateral intravitreal injection of GS010: a randomized, double-masked, placebo-controlled trial in subjects affected with G11778A ND4 leber hereditary optic neuropathy for up to one year. GenSight Biologics
Humans
human wild-type MT-NDA (mitochondrial NADH Dehydrogenase 4)
