Retinal Research Community Urges Johnson & Johnson to Seek Regulatory Approval for its XLRP Gene Therapy

The Foundation Fighting Blindness, along with the investigators participating in the Phase 3 LUMEOS clinical trial for the X-linked retinitis pigmentosa (XLRP) gene therapy bota-vec, strongly urge Johnson & Johnson (J&J) to file a biologics license agreement (BLA) for bota-vec with the US Food &Drug Administration (FDA) as well as regulators in other regions, including the European Medicines Agency (EMA) and Medicines and Healthcare products Regulatory Agency (MHRA) in the UK. The request comes as a result of J&J’s and patients’ reports of meaningful vision improvements in LUMEOS for people treated bilaterally with bota-vec.

“The vision improvements in LUMEOS have been life changing for many patients. Both objective measures and subjective reports from patients demonstrate clear and meaningful efficacy,” says Todd Durham, PhD, senior vice president of clinical outcomes and research at the Foundation Fighting Blindness. “The Foundation and trial investigators feel it is incumbent upon J&J to urgently pursue regulatory approval to make the vision-restoring treatment available to the global XLRP community.”

Some of the many comments from and about patients treated in a bota-vec trial:

“Our son Matthew received gene therapy surgery in March 2024 through the LUMEOS clinical trial. Since then, we’ve seen meaningful improvements in his vision—especially in low-light and nighttime settings. Tasks that were once frustrating or challenging, like moving around safely in dim rooms or walking outdoors after sunset, have become noticeably easier for him. These changes have restored a sense of independence and confidence in his daily life.”

"I saw stars for the first time and clouds at night! It was amazing.”

“The treatment has dramatically improved my eyesight. I used to need a cane at night, and sometimes during the day as well, and now I no longer need to use a cane.”

“The improvement in my vision post-surgery has been nothing short of remarkable. Having personally experienced the significant benefits to my treated eye; I would do anything within my power to have the same life-changing opportunity extended to my untreated eye.”

Summary of the 52-week LUMEOS results

In the Phase 3 clinical trial, many treated patients showed improvements in vision as measured by low luminance visual acuity (LLVA) testing, static perimetry (visual fields), and/or visually guided mobility assessment (VMA). Improvements in LLVA were most significant with 46 percent of patients (25/55) having at least 10 letters (two lines on an eye chart) of improvement compared to 7 percent (2/30) in the control (deferred treatment) group. J&J also reported that 40 percent (22/55) of treated patients showed improvement in two or more endpoints, with no patients (0/30) in the control group having improvement in at least two endpoints.

LUMEOS investigators urging J&J to seek regulatory approval for bota-vec:

Isabelle Audo, MD, PhD, Institut de la Vision
Prof. James Bainbridge, Moorfields Eye Hospital
Eyal Banin, MD, PhD, Hadassah Medical Center
Shyamanga Borooah, MBBS, PhD, University of California, San Diego
Prof. Camiel J.F. Boon, MD, PhD, Amsterdam University Medical Center
Dr. Ayuso García Carmen, Jiménez Díaz Foundation
Jason Comander, MD, PhD, Mass Eye and Ear, Harvard University
Kunal Dansingani, MD, University of Pittsburgh Medical Center
David Gilmour, MD, NHS Greater Glasgow and Clyde
Elise Héon, MD, Hospital for Sick Kids, Toronto
Dr. Tran Hoai Viet, University de Lausanne, Hopital Ophthalmique, Jules-Gonin
Prof. Carel Hoyng, MD, PhD, Radboud University Medical Center
Nieraj Jain, MD, Emory University
Dr. Kamron Khan, Leeds Teaching Hospital, NHS Trust
Rachel Huckfeldt, MD, PhD, Mass Eye and Ear, Harvard University
Christine Kay, MD, Vitreo Retinal Associates, Gainesville
Prof. Line Kessel, PhD, University of Copenhagen
Prof. Bart Leroy, MD, PhD, Ghent University Hospital
Vinit Mahajan, MD, PhD, Stanford University
Dr. Roly Megaw, NHS Lothian, University of Edinburgh
Manjit Mehat, MB ChB PhD, NHS Lothian, University of Edinburgh
Prof. Michel Michaelides, BSc MB BS MD(Res), Moorfields Eye Hospital
Raj Mukherjee, Leeds Teaching Hospital, NHS Trust
Aaron Nagiel, MD, PhD, Children's Hospital Los Angeles
Prof. José-Alain Sahel, MD, University of Pittsburgh Medical Center
Dr. Blanca Garcia Sandoval, Hospital Universitario Fundación Jiménez Díaz
Hendrik Scholl, MD, Medical University of Vienna
Prof. Francesca Simonelli, Università degli Studi della Campania Luigi Vanvitelli
Lejla Vajzovic, MD, Duke University
Thomas Wubben, MD, PhD, Kellogg Eye Center, University of Michigan