XLRP Clinical Trial Result

The biggest news of the conference, which has already been shared with Retina Australia members, was that sadly the Janssen Pharmaceuticals RPGR gene therapy trial did not meet its primary endpoints, although there was some positive news.

Janssen, a division of Johnson & Johnson, recently completed clinical trials for their gene therapy targeting X-linked retinitis pigmentosa (XLRP) caused by mutations in the RPGR gene. The therapy, known as botaretigene sparoparvovec (formerly JNJ-81201887), aimed to deliver a functional copy of the RPGR gene to retinal cells. Despite initial promise, the Phase 3 LUMEOS trial failed to meet its primary endpoint – an assessment of vision-guided mobility. The study involved 95 patients treated over a 52 week period. However, there were signs of efficacy in other vision measures, and some of the patients did very well after the treatment. As such, Janssen will continue to analyse the data and determine their next steps.

Despite this disappointing news, many exciting advances were reported at the meetings. Lauren, Ceecee and Erica have provided the following update from the conference, including summaries on gene editing, RNA therapies, cell therapies, and drug treatments, all aimed at preserving or restoring vision in people with IRDs.