Atsena completes dosing in part A of X-linked retinoschisis gene therapy trial

Atsena Therapeutics completed patient dosing in part A of the phase 1/2 LIGHTHOUSE clinical trial investigating a gene therapy designed for the treatment of X-linked retinoschisis, according to a press release.

Part A of the trial is evaluating three dose levels of ATSN-201, a subretinal injection, in nine adults with X-linked retinoschisis (XLRS). The therapy uses AAV.SPR, the company’s novel spreading capsid, to reach therapeutic levels of gene expression in photoreceptors in the central retina, according to the release.

The open-label, dose-escalation, dose-expansion LIGHTHOUSE trial is designed to evaluate the safety and tolerability of ATSN-201 in male patients aged 6 years and older who have XLRS. As Healio previously reported, the therapy received both orphan drug designation and rare pediatric disease designation from the FDA.

“This marks a significant milestone for patients with XLRS as we push to bring a therapeutic option to individuals that otherwise have no approved treatment,” Patrick Ritschel, CEO of Atsena, said in the release “It also represents potential for individuals living with other inherited retinal diseases that could benefit from our novel capsid. We look forward to the insights gained from this trial as we pioneer new approaches to ocular gene therapy.”

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