MeiraGTx Acquires XLRP Gene Therapy Bota-Vec, Moves Toward Regulatory Filing

MeiraGTx has acquired botaretigene sparoparvovec (bota-vec), a gene therapy for X-linked retinitis pigmentosa (XLRP), from Johnson & Johnson. The company plans to file for regulatory approval in the U.S., Europe, and Japan as quickly as possible, with the potential for a commercial launch in 2027.

Bota-vec is a one-time gene therapy delivered by injection into both eyes. It uses an adeno-associated virus (AAV) to deliver a functional copy of the RPGR gene — the gene most commonly mutated in XLRP — directly to the retina. XLRP is a rare inherited retinal disease that causes progressive vision loss beginning in childhood, often leading to complete blindness by the third decade of life. As an X-linked disease, XLRP predominantly affects males, though female carriers may have varying degrees of disease. There are currently no approved treatments.

Data from Johnson & Johnson’s Phase 3 LUMEOS study, which enrolled 95 patients treated in both eyes, showed meaningful improvements across multiple measures of vision. While the study’s primary endpoint — a functional vision maze test — did not reach statistical significance, secondary endpoints told a strong story. Retinal sensitivity improved significantly across all measures. In terms of visual acuity, 45% of treated patients gained more than 10 letters in low-light conditions, a nd 20% gained more than 15 letters. Patients also reported meaningful improvements in mobility, dim light function, and emotional wellbeing. Notably, 40% of treated patients showed improvement in two or more distinct domains of vision, compared to none in the untreated control group. The safety profile was consistent with earlier trials, with no new safety concerns identified.

Foundation Fighting Blindness CEO Jason Menzo commented, “For patients living with X-linked retinitis pigmentosa, the need for treatment options is clear and urgent. The data from the LUMEOS Phase 3 study of bota-vec, reflected in both objective measures and patient-reported outcomes, point to real improvements in vision.”

MeiraGTx is well positioned to move quickly toward filing: as the commercial manufacturer of bota-vec, the company has already completed the manufacturing validation steps required for regulatory submissions. The FDA has granted bota-vec Fast Track and Orphan Drug designations, and regulators in the European Union have granted Priority Medicines (PRIME) and Orphan Drug designations — all of which are designed to help expedite the review process.

For the RPGR-associated XLRP community, this acquisition is a meaningful step forward. With regulatory filings expected to begin soon and a potential launch in 2027, bota-vec may be on a path to becoming the first approved treatment for this blinding disease. For more information, visit www.MeiraGTx.com.