“Part B of our LIGHTHOUSE study is delivering exactly as we expected — a favorable safety profile, schisis resolution, and functional improvements at six months that closely replicate what we observed at the same time point in Part A of the study. Foveal schisis closure was observed in four of six treated adults, and there was no structural change observed in untreated control subjects or untreated contralateral eyes. The kinetics of microperimetry response across Cohort 4 mirror what we saw in Part A of the study. No serious adverse events were observed in any Part B cohort, including in our pediatric patients,” said Shannon Boye, PhD, Co-Founder and Chief Scientific Officer of Atsena.

Read more about Atsena Presents Positive Interim Six-Month Results From Part B of Phase 1/2/3 LIGHTHOUSE Trial Evaluating ATSN-201 in Patients With X-Linked Retinoschisis.

Read more about Genezen and Atsena Therapeutics Announce Strategic Commercial Manufacturing Partnership.

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 dosing is complete in patients enrolled across all adult and pediatric cohorts in Part B of the Phase I/II/III Lighthouse Trial evaluating subretinal injection of ATSN-201 for the treatment of X-linked retinoschisis (XLRS). The Company plans to initiate enrollment of the pivotal Part C cohort of patients in the first quarter of 2026.

Read more about Atsena Completes Dosing in Part B of the Phase I/II/III LIGHTHOUSE Trial Evaluating ATSN-201 To Treat X-Linked Retinoschisis and Announces Plans for Initiation of Pivotal Part C Cohort.

Atsena Therapeutics Announces Dosing Complete for Adults in Part B of the Phase I/II/III LIGHTHOUSE Trial Evaluating ATSN-201 to Treat X-Linked Retinoschisis.

“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.

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 granted Regenerative Medicine Advanced Therapy (RMAT) designation for ATSN-201 for the treatment of X-linked retinoschisis (XLRS). ATSN-201, a best-in-class gene therapy product candidate, leverages AAV.SPR, the company’s novel spreading capsid, to achieve therapeutic levels of gene expression in photoreceptors of the central retina while avoiding the surgical risks of foveal detachment.

“We’re honored that the FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation to ATSN-201, further underscoring its potential to address the urgent, unmet need in XLRS—a rare inherited retinal disease with no approved treatments,” said Patrick Ritschel, Chief Executive Officer of Atsena Therapeutics.

“We are pleased that the FDA has granted Fast Track designation to ATSN-201, reinforcing its potential to address the significant unmet need in XLRS, a rare inherited retinal disease with no approved treatments,” said Patrick Ritschel, Chief Executive Officer of Atsena Therapeutics. “This designation, along with the previously granted Orphan Drug and Rare Pediatric Disease designations, marks an important milestone in advancing the development of ATSN-201. The Atsena team remains dedicated to developing transformative gene therapies and improving the quality of life of individuals suffering from XLRS and other inherited retinal diseases.”

Delivering AAV gene therapy to the retina to treat a rare inherited retinal disease is not without risk.

For one, using conventional gene therapy vectors to target genes to the central retina (the area where therapy is needed) requires detaching that region of the retina. Beyond that, transduction doesn’t happen much beyond the injection bleb, which can limit therapeutic effect.