Atsena reports early efficacy signals for gene therapy ATSN-201 in X-linked retinoschisis trial
Atsena Therapeutics presented data from the early parts of its Lighthouse gene therapy trial for X-linked retinoschisis (XLRS) at the American Academy of Ophthalmology meeting.
The trial administered the ATSN-201 gene therapy to one eye of 18 patients, with three untreated controls. Half of the treated eyes met the microperimetry response criteria, the primary endpoint for the phase-3 portion. Six of nine patients receiving the phase-3 dose responded, while none of the controls did.
Based on these results, Atsena selected the lowest dose for the upcoming phase-3 segment, citing consistent efficacy and better tolerability. The company plans to continue the study toward a potential regulatory filing in 2028.
This writeup was produced by pharmadog from original reporting by Fierce Biotech.
Original headline: “Atsena’s Lighthouse gene therapy readout illuminates eye disease efficacy”
read at Fierce Biotech ↗
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