Atsena Reports LIGHTHOUSE data at ARVO - Takeaways - MDSpire

Atsena Reports LIGHTHOUSE data at ARVO

Atsena Therapeutics announced positive clinical data results from Part A of the LIGHTHOUSE study, a phase 1/2 clinical trial evaluating subretinal injection of ATSN-201 for the treatment of X-linked retinoschisis (XLRS). The data were presented at the 2025 Association for Research in Vision and Ophthalmology meeting in Salt Lake City, Utah.

  • May 1, 2025

  • 3 min

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    Atsena Therapeutics presented positive data from Part A of the LIGHTHOUSE study at the 2025 ARVO meeting.

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    ATSN-201, a gene therapy for X-linked retinoschisis, showed good safety and efficacy in a phase 1/2 clinical trial.

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    The therapy utilizes AAV.SPR to deliver gene expression in retinal photoreceptors while minimizing surgical risks.

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    Part A of the study involved 9 adult patients, with no serious adverse events related to ATSN-201 reported.

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    Enrollment is ongoing for Part B, which will include additional adults and pediatric patients for further evaluation.

Original Source(s)

Atsena Reports LIGHTHOUSE data at ARVO

  • May 1, 2025

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