Part B of phase 1/2 X-linked retinoschisis gene therapy trial initiated

Atsena Therapeutics initiated part B of the phase 1/2 LIGHTHOUSE study investigating ATSN-201 for the treatment of X-linked retinoschisis, according to a press release.

The multicenter clinical trial will include nine adult patients and three pediatric patients with X-linked retinoschisis. At the recommendation of a data monitoring committee, patients will be dosed with ATSN-201 1E11 vg/mL, “a concentration that offered the optimal balance of tolerability and efficacy based on preliminary clinical results,” the release said.

The adult cohort will be divided into low-volume, high-volume and control arms, and the pediatric cohort will be dosed after evaluating preliminary data from the adult cohort. Part B will continue to assess the safety and efficacy of the gene therapy product candidate as measured through microperimetry, visual acuity and macular structure.

“The functional and structural improvements seen in part A validate our novel AAV.SPR spreading capsid,” Patrick Ritschel, CEO of Atsena, said in the release. “Importantly, part B will enroll both adult and pediatric participants. The rare pediatric disease designation granted for ATSN-201 highlights the unmet need for a treatment, and we remain focused on advancing a therapeutic option for these patients.”

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