B/BE/26/BVW2 - Information sheet for the public (prepared by the notifier)Public information according to the provisions of Directive 2001/18/EC
Long title
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)
Date receipt dossier
EU record number
B/BE/26/BVW2
EudraCT number
2025-522207-15-01
Company / Sponsor
AAVantgarde Bio UK Ltd.
Phase
I/II
Treated organism
Humans
Indication category
Eye degenerative disease
Disease
Stargardt Disease (STGD1)
Therapeutic approach
Gene therapy
Genetic modification
Non-replicating recombinant dual vector derived from adeno-associated virus AAV8 carrying functional copies of the ABCA4 gene.
Method of transfer of nucleic acid of interest
Non-replicating recombinant vector derived from adeno-associated virus AAV8
Administered biological material
Genetically modified virus
Route of administration
Subretinal
Locations in Belgium
UZ Gent
Nr of subjects
Up to 15 patients overall and 3 patients in Belgium
Foreseen duration
From 19 May 2026 till 29 Oct 2027
Type of procedure
Contained use and Deliberate release
Current status
Authorized