Hope in Focus: 24-Month Results from the SKYLINE Gene Therapy Trial for X-Linked Retinitis Pigmentosa

At A Race Against Blindness, we are committed to following every development that brings hope to families living with rare retinal diseases. One exciting update came from the 2025 American Society of Retina Specialists (ASRS) annual meeting in Long Beach, California, where researchers shared encouraging results from the Phase 2 SKYLINE trial of a promising new therapy for X-linked retinitis pigmentosa (XLRP) . What is X-Linked Retinitis Pigmentosa? XLRP is a rare, inherited retinal disease caused by mutations in the RPGR gene . It primarily affects males and leads to progressive vision loss, often beginning in childhood or adolescence. Over time, XLRP can cause severe blindness. Today, there are no FDA-approved treatments — which makes gene therapy research especially critical. The SKYLINE Phase 2 Trial The SKYLINE trial investigated laruzova , a full-length RPGR gene therapy designed to target the underlying genetic cause of XLRP. Study Design Participants: Males ages 8–50 with a confirmed RPGR mutation Inclusion criteria: Measurable microperimetry sensitivity (a way of measuring ho