Atsena Therapeutics Initiates Dosing in Second Cohort of Phase 1/2 Trial Evaluating XLRS Gene Therapy

Dosing has been initiated in the second cohort of the LIGHTHOUSE study, a phase 1/2 clinical trial evaluating subretinal injection of ATSN-201 for the treatment of X-linked retinoschisis (XLRS), according to Atsena Therapeutics.

ATSN-201, a gene therapy product candidate, leverages AAV.SPR, Atsena'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. ATSN-201 was well tolerated in all three XLRS patients in the first cohort and no serious adverse events were reported. The Data and Safety Monitoring Board recommended proceeding with the second cohort, in which patients will receive the next dose level of ATSN-201.

"We are encouraged by the excellent safety observed with the low dose of ATSN-201 in the LIGHTHOUSE study, which represents the first clinical utilization of AAV.SPR. Additionally, initial indications of structural and functional improvement observed within and beyond the injection areas suggest successful lateral spread of the vector,” Kenji Fujita, MD, Chief Medical Officer of Atsena Therapeutics, said in a company news release. “Advancing into the second cohort brings us one step closer to potentially bringing a much-needed one-time treatment to patients with XLRS, an inherited retinal disease that causes progressive vision loss and can lead to blindness.”

The LIGHTHOUSE study is a phase 1/2, open-label, dose-escalation and dose-expansion clinical trial evaluating the safety and tolerability of ATSN-201 in male patients ages 6 and older with a clinical diagnosis of XLRS caused by pathogenic or likely pathogenic mutations in RS1. Enrollment in the LIGHTHOUSE study is ongoing. For more information, visit ClinicalTrials.gov (Identifier: NCT05878860).