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Gene Therapy Trial Report

Summary

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


NCTID NCT05878860 (View at clinicaltrials.gov)
Description
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
Enrollment Count 21 (ESTIMATED)
Results Posted Not Available

Therapy Information


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
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)

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2023-05-12
Completion Date 2029-10
Last Update 2025-02-19

Participation Criteria


Eligible Age >=6 Years
Standard Ages Child, Adult, Older adult
Sexes Eligible for Study MALE
Eligibility Criteria
Inclusion Criteria: 1. Age ≥ 18 for Cohorts 1 through 3, and age ≥ 6 years and \< 18 years for Cohort 4. 2. Male patients with clinical diagnosis of XLRS caused by mutations in RS1. 3. Best corrected visual acuity (BCVA) in study eye of 34 to 73 Early Treatment Diabetic Retinopathy Study (ETDRS) letters (corresponding to a Snellen acuity of 20/200 to 20/40). Exclusion Criteria: 1. Pre-existing eye conditions in the study eye that would contribute significantly to an increased risk of visual loss from a subretinal injection. 2. Any intraocular surgery (including laser treatment) in the study eye within 6 months prior or any intraocular surgery anticipated in the study eye during the first 12 months of the study. 3. Treatment in a prior ocular gene or cell therapy study.
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 4
Locations United States

Regulatory Information


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

Resources/Links