Search the database for deliberate release of GM medicinal products
EU record number
Title
Company / Sponsor
Treated organism
Genetic modification
B/BE/26/BVW2
An Open-label, Multicenter, Two Part, Ascending Dose Followed by a Controlled Trial to Assess the Safety and Efficacy of a Subretinal Administration of AAVB-039 in Participants with Stargardt Disease (STGD1) (CELESTE) AAVantgarde Bio UK Ltd.
Humans
Non-replicating recombinant dual vector derived from adeno-associated virus AAV8 carrying functional copies of the ABCA4 gene.
Only notified under the "contained use" procedure. Dossier submitted on 25 March, 2025
.
EXACT 2 is a randomized, multicenter, double-blind, placebo-controlled Phase 2b trial designed to evaluate the safety, feasibility, and efficacy of percutaneous endovascular catheter-facilitated intramyocardial administration of XC001 in patients with ref XyloCor Therapeutics
Humans
VEGF
B/BE/22/BVW5
A two-part, open-label systemic gene delivery study to evaluate the safety and expression of RO7494222 (SRP-9001) in subjects under the age of four with Duchennne Muscular Dystrophy (ENVOL) F. Hoffmann-La Roche
Humans
Non-replicating recombinant vector derived from adeno-associated virus AAV, serotype rh74, lacking all AAV viral genes and carrying the human micro-dystrophin (hMicro-Dys) gene
