Jun 17, 2025

FBC Supports Path Forward for XLRP Treatment

Fighting Blindness Canada Urges Johnson & Johnson to Continue Advancing Gene Therapy for XLRP
Fighting Blindness Canada is urging Johnson & Johnson and MeiraGTx to continue pursuing regulatory approval for botaretigene sparoparvovec (bota-vec), a gene therapy for X-linked retinitis pigmentosa (XLRP). This potential treatment has offered new hope to individuals and families affected by a rare and progressive genetic eye disease that currently has no approved therapies

The Phase 3 LUMEOS trial has shown encouraging signs of benefit with many treated patients showing vision improvements as measured by low luminance visual acuity (LLVA) testing, static perimetry (visual fields), and/or visually guided mobility assessment (VMA). Overall, 22 out of 55 (40%) demonstrated improvements in two or more measures of visual function, compared to none in the control group. These outcomes reflect real, meaningful gains that could change lives for people living with XLRP.

“This is a pivotal moment for people living with XLRP. The meaningful improvements seen in multiple patients are cause for optimism. We urge Johnson & Johnson to move forward with regulatory engagement and for decision-makers to consider the full scope of benefit that this therapy may offer.”
— Larissa Moniz PhD, Director of Research and Mission Programs, Fighting Blindness Canada

XLRP primarily affects males from a young age, leading to significant vision loss in adolescence or early adulthood. For those living with the condition, even modest slowing of vision loss can profoundly impact quality of life.

“XLRP has been slowly taking my vision since the day I was born… Today, I have about 5 degrees of visual field left and it’s just a matter of time before my narrow tunnel closes in and I will go totally blind. But, the J&J XLRP gene therapy could change that outcome, which would be truly life changing. I don’t need a miracle cure and get all of my vision back, I just want to stop going blind. I want nothing more than to be able to continue to see my daughter grow up, that would mean the world to me.”
— Bernie Zinck, XLRP patient advocate, Nova Scotia

Leading experts in inherited retinal disease have also voiced strong support for moving this therapy forward, pointing to the extensive and clinically relevant data generated across all phases of testing.

“XLRP is a devastating progressive form of vision loss. Young men cannot see in the dark or when coming inside from the outside bright light and they also have tunnel vision. Patients having received botaretigene sparoparvovec are more confident as they can navigate better in different light settings. For teenagers and young adults this is life changing. A small improvement can make the world of difference to the quality of life of patients. We have many Canadian patients losing sight every day, waiting for this treatment to be approved.”
— Dr. Elise Heon, SickKids Hospital, Ontario

“The clinically meaningful data published and presented for the Phase 1, 2 and 3 clinical trials of the Johnson & Johnson gene therapy for X-linked RP associated with RPGR, are truly game changing for patients and their families. We need to accelerate approvals of such treatments that address unmet need in young children and working age adults, to allow them to have the fullest personal and professional lives, and also address the enormous emotional and psychological toll these rare disease have on patients and their families.”
— Prof. Michel Michaelides, Moorfields Eye Hospital, UK

Fighting Blindness Canada stands alongside the XLRP community in calling for a continued path forward for bota-vec. We recognize Johnson & Johnson’s ongoing efforts to evaluate the data and encourage them to pursue all regulatory options. At the same time, we ask regulatory agencies to consider the totality of the clinical evidence and the urgent, unmet need in this rare disease.

We will continue to work with patients, researchers, and our advocacy partners to ensure the voices of those impacted by XLRP are heard.

Learn more about clinical trials.