Objective:
To evaluate the safety and efficacy of ATSN-201, an investigational gene therapy for X-linked retinoschisis (XLRS), over 18 months.
Approach:
- Study Design: Phase 1/2 LIGHTHOUSE trial involving nine adults receiving subretinal injections of ATSN-201.
Key Findings:
- Foveal schisis closure was maintained in 7 of 9 treated eyes at 18 months and was not observed in untreated eyes.
- Six treated eyes showed an improvement of at least 7 dB on microperimetry, compared with none of the untreated eyes.
- Seven treated eyes had gains of at least 10 letters in either best-corrected or low-luminance visual acuity, compared to 1 untreated eye.
Interpretation:
ATSN-201 demonstrated sustained structural and functional improvements in patients with XLRS without significant safety concerns.
Limitations:
- Small sample size of nine adults.
- Results are preliminary and from a phase 1/2 study.
Conclusion:
Enrollment is ongoing for the pivotal Part C cohort, with completion expected in the first quarter of 2027 and results anticipated in the first half of 2028.
Sources:
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