ATSN-201 Gene Therapy in RS1-Associated X-linked Retinoschisis

NCTID	NCT05878860 (View at clinicaltrials.gov)
Description	This study will evaluate the safety and tolerability of ATSN-201 in male subjects ≥ 6 years of age with RS1-associated X-linked retinoschisis (XLRS). (Show More)
Development Status	Active
Indication	X-Linked Retinoschisis
Disease Ontology Term	DOID:0060763
Compound Name	ATSN-201
Compound Description	AAV.SPR-hGRK1-hRS1syn
Sponsor	Atsena Therapeutics Inc.
Funder Type	Industry
Recruitment Status	Recruiting (Click here for study contact information)
Enrollment Count	21 (ESTIMATED)
Results Posted	Not Available

Target Gene/Variant	RS1
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Subretinal
Drug Product Type	Viral vector
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	AAV.SPR
Editor Type	none
Dose 1	1.5E10 vg/eye
Dose 2	3.0E10 vg/eye
Dose 3	5.0E10 vg/eye
Dose 4	1.5E10 vg/eye (150ul)
Dose 5	2.3E10 vg/eye (225ul)

No.of Trial Sites	4
Locations	United States

Has US IND	True
FDA Designations	Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy
Recent Updates	Pediatric dosing expected to begin Q4 2025; pivotal cohort expected to begin enrolling in Q1 2026