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)
Indication	Retinoschisis, X-linked
Compound Name	ATSN-201 (AAV.SPR-hGRK1-hRS1syn)
Sponsor	Atsena Therapeutics Inc.
Funder Type	Industry
Status	Recruiting
Enrollment Count	21

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
Dose 1	1.5 x 10^10 vg/eye
Dose 2	5.0 x 10^10 vg/eye
Dose 3	1.7 x 10^11 vg/eye
Dose 4
Dose 5

No.of Trial Sites	4
Locations	United States

Has US IND	True
Recent Updates	FDA granted Rare Pediatric Disease Designation

Atsena Therapeutics – Atsena Therapeutics Receives Rare Pediatric Disease Designation from the U.S. FDA for ATSN-201 Gene Therapy to Treat X-linked Retinoschisis (atsenatx.com)