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)

Study 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)
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: 
3 juin 2026
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: 
Under evaluation

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