6 May, 2025
The LUMEOS study was sponsored by Janssen Research & Development, LLC (a division of Johnson & Johnson). This was a Phase 3 randomised, controlled study of an investigational adeno-associated virus vector type 5 (AAV5) gene therapy, botaretigene sparvoparvovec (bota-vec). The study was for the treatment of X-linked retinitis pigmentosa (XLRP) associated with disease causing sequence variants in the retinitis pigmentosa GTPase regulator (RPGR) gene.
According to reports from the Retina Therapeutics Innovation Summit on 2nd May in Salt Lake City, Utah (3), a spokesperson for the LUMEOS study has advised that unfortunately the study did not meet its primary endpoint that compared the functional vision of patients treated before and at 52 weeks after gene therapy delivery. The study’s primary outcome measure was to evaluate a change from baseline (i.e., prior to treatment) and then 52 weeks after treatment of the participant to navigate through a Vision-guided Mobility Assessment (VMA) maze.
In a statement from Johnson & Johnson 5 May 2025, they said “The Phase 3 LUMEOS study did not meet its primary endpoint but showed improvement in the majority of secondary endpoints. Johnson & Johnson is working to actively understand the totality of the data and its clinical relevance.”
https://retinaaustralia.com.au/x-linked-retinitis-pigmentosa-rpgr-gene-therapy-study-update/
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