B/BE/26/BVW4 - Information sheet for the public (prepared by the notifier)Public information according to the provisions of Directive 2001/18/EC
Long title
A Phase 1/2/3, Open-Label, Dose Escalation, Dose Expansion, and Randomized, Controlled Study to Evaluate the Safety and Efficacy of ATSN-201 Gene Therapy in Subjects with RS1-Associated X-linked Retinoschisis (LIGHTHOUSE)
Date receipt dossier
EU record number
B/BE/26/BVW4
EudraCT number
2026-525886-38-00
Company / Sponsor
ASTENA Therapeutics
Phase
I/II/III
Treated organism
Humans
Indication category
Eye degenerative disease
Disease
RS1-Associated X-linked Retinoschisis
Therapeutic approach
Gene therapy
Genetic modification
Non-replicating recombinant vector derived from adeno-associated virus AAV44.9 carrying functional copies of the human retinoschisin (hRS1) gene.
Method of transfer of nucleic acid of interest
Non-replicating recombinant vector derived from adeno-associated virus AAV44.9
Administered biological material
Genetically modified virus
Route of administration
Subretinal
Locations in Belgium
UZ Gent
Nr of subjects
Up to 106 patients overall and 6 patients in Belgium
Foreseen duration
From 25th October 2026 to 25th September 2033
Type of procedure
Contained use and Deliberate release
Current status
Assessed