5 Key Takeaways

• 1

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

• 2

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

• 3

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

• 4

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

• 5

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