In May 2025, Johnson & Johnson (J&J) announced that though its emerging gene therapy bota-vec for X-linked retinitis pigmentosa (XLRP) improved vision for many patients in its Phase 3 clinical trial, the gene therapy didn’t meet its primary endpoint. Not meeting a primary endpoint in a clinical trial is not good news even when overall results are favorable.
So, the question became: Will J&J seek regulatory approval for bota-vec or perhaps continue clinical development for it? Many of us in the inherited retinal disease (IRD) community were not optimistic. We were afraid bota-vec development was finished and the treatment would never make it out to patients who so desperately needed it.
But today I am pleased to say that hope is alive for bota-vec. On April 16, 2026 ⎯ nearly a year later ⎯ MeiraGTx, the original developer of bota-vec, announced it is acquiring the bota-vec license from J&J. MeiraGTx plans to seek regulatory approval for the gene therapy in the US and EU. If approved ⎯ and that’s still a big if ⎯ bota-vec could be available to patients in 2027.
We applaud MeiraGTx ⎯ a Hope in Focus partner and developer of a vision-restoring gene therapy for LCA4 (AIPL1 mutations) ⎯ for giving new life to bota-vec. It was a bold move, no doubt. And, we applaud the many advocates in the IRD community ⎯ including researchers, patient groups, and families ⎯ for being strong voices in support of bota-vec advancement. Our voices were heard.
The important take-away message for all of us in the IRD and LCA communities is to stay tenacious, stay resilient, and continue to do our best in moving the research forward.
And never forget that hope is our superpower.