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ATSN-201 Gene Therapy in RS1-Associated X-linked Retinoschisis
Sponsor: Atsena Therapeutics Inc.
Summary
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).
Official title: A Phase 1/2, Open-Label, Dose Escalation and Dose Expansion Study to Evaluate the Safety and Tolerability of ATSN-201 Gene Therapy in Male Subjects With RS1-Associated X-linked Retinoschisis
Key Details
Gender
MALE
Age Range
6 Years - Any
Study Type
INTERVENTIONAL
Enrollment
21
Start Date
2023-08-22
Completion Date
2029-10
Last Updated
2025-02-19
Healthy Volunteers
No
Conditions
Interventions
ATSN-201
AAV.SPR-hGRK1-hRS1syn
Locations (4)
Children's Hospital of Los Angeles
Los Angeles, California, United States
Bascom Palmer Eye Institute
Miami, Florida, United States
Oregon Health Sciences University
Portland, Oregon, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States