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
B/BE/19/BVW2
A Phase 3 Open-Label, Single-Arm Study To Evaluate The Efficacy and Safety of BMN 270, an Adeno-Associated Virus Vector–Mediated Gene Transfer of Human Factor VIII in Hemophilia A Patients with Residual FVIII Levels Receiving Prophylactic FVIII BioMarin Pharmaceutical Inc.
Humans
Recombinant adeno-associated virus serotype 5 (AAV5) containing a gene cassette expressing the human coagulation factor VIII (FVIII) gene
Only notified under the "contained use" procedure. Dossier submitted on 4 October, 2018
.
A Phase 3, Randomized, Double-blind, Placebo-controlled study to Determine the Efficacy and Safety of CMB305 in Unresectable Locally-advanced or Metastatic NY-ESO-1 + Synovial Sarcoma Subjects Following First-line Systemic Anti-cancer Therapy Immune Design
Humans
Replication deficient lentiviral vector encoding NY-ESO-1 cancer testis antigen
