Atsena Therapeutics Announces Alignment With FDA on Regulatory Pathway to Approval for ATSN-201 in X-Linked Retinoschisis (XLRS)

UF startup Atsena Therapeutics, a clinical-stage gene therapy company focused on using the life-changing power of genetic medicine to reverse or prevent blindness, announced that the U.S. Food and Drug Administration (FDA) has agreed to the expansion of the company’s ongoing Phase 1 / 2 LIGHTHOUSE study of ATSN-201 into a continuous Phase 1 / 2 / 3 trial, enabling it to serve as a pivotal trial to support a Biologics License Application (BLA) submission for the treatment of X-linked retinoschisis (XLRS). The BLA submission is anticipated in early 2028.

“This regulatory milestone marks another significant step toward delivering a potentially first- and best-in-class gene therapy for patients living with XLRS,” said Patrick Ritschel, MBA, Chief Executive Officer of Atsena Therapeutics.

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