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/25/BVW3
Phase 3, Open-label, Single-dose Study of CSL222 in Adolescent Male Subjects (≥ 12 to < 18 Years of Age) with Severe or Moderately Severe Hemophilia B CSL Behring LLC
Humans
Recombinant adeno associated viral vector containing the codon-optimised human PADUA factor IX (FIX) gene
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
