Search the database for deliberate release of GM medicinal products
EU record number
Title
Company / Sponsor
Treated organism
Genetic modification
B/BE/24/BVW5
Phase 3, open-label, single-arm study to evaluate efficacy and safety of FIX gene transfer with PF-06838435 (rAAV-Spark100-hFIX-R338L) in adult male participants with moderately severe to severe hemophilia B (FIX:C ≤2%) (BeneGene-2) Pfizer
Humans
Recombinant adeno associated viral vector containing the codon-optimised human factor IX (FIX) minigene (R338L variant)
Only notified under the "contained use" procedure. Dossier submitted on 5 June 2024
.
A Phase 2, Open-Label, Randomized, Multicenter Study of KYV 101, an Autologous Fully Human Anti-CD19 Chimeric Antigen Receptor T-Cell (CD19 CAR T) Therapy, in Subjects with Refractory Primary and Secondary Progressive Multiple Sclerosis (KYSA-7) Kyverna Therapeutics, Inc.
Humans
CD19 CAR
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
B/BE/20/BVW6
Dossier withdrawn by the notifier Pfizer
Humans
Non-replicating recombinant vector derived from adeno-associated virus AAV6 serotype, containing a codon-optimised version of the human Factor VIII gene
B/BE/20/BVW4
A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy Pfizer
Humans
Non-replicating recombinant vector derived from adeno-associated virus AAV9, lacking all AAV viral genes and containing a miniaturized version of the human dystrophin gene
