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