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 22 May, 2023
.
A randomized, open-label, Phase 2 study evaluating lymphodepletion with fludarabine (F), cyclophosphamide (C), and ALLO-647 (A) vs FC alone, in subjects with R/R LBCL receiving ALLO-501A allogeneic CAR T cell therapy Allogene therapeutics
Humans
CD19 CAR + knockouts CD52 and TRAC via TALEN technology
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)
