April 16, 2026 —
Johnson & Johnson (J&J) has entered into an asset purchase agreement with MeiraGTx, transferring full rights to the investigational gene therapy bota-vec for the treatment of X-linked retinitis pigmentosa (XLRP), a rare inherited retinal disease with no approved therapies. The transaction includes a $25 million upfront payment, along with future regulatory and commercial milestones and high double-digit royalties on global net sales beginning in 2029.
Bota-vec (botaretigene sparoparvovec) is an AAV-based gene therapy targeting mutations in the RPGR gene, a leading cause of XLRP. The therapy has demonstrated encouraging clinical activity in the Phase 3 LUMEOS study, including improvements across multiple functional and patient-reported vision endpoints. While the primary endpoint did not reach statistical significance, secondary endpoints showed clinically meaningful benefits, including gains in low luminance visual acuity and retinal sensitivity.
According to MeiraGTx, the company is well-positioned to rapidly advance regulatory filings, having served as the commercial manufacturer for the program and already completed process performance qualification (PPQ). The company plans to initiate biologics license application (BLA) and marketing authorization application (MAA) submissions across the U.S., Europe, and Japan in the near term, with a potential commercial launch targeted for 2027.
The asset reacquisition reflects strong support from the clinical and patient community. Investigators involved in the LUMEOS study have highlighted meaningful real-world improvements in vision, while advocacy organizations such as the Foundation Fighting Blindness have publicly supported regulatory advancement based on the therapy’s potential to address a significant unmet need.
XLRP affects more than 20,000 patients across the U.S. and Europe and typically leads to progressive vision loss and eventual blindness by early adulthood. With no existing treatment options, bota-vec represents a potentially disease-modifying therapy for this underserved population. MeiraGTx also indicated that this program, alongside its AAV-hAQP1 program for radiation-induced xerostomia, could position the company to transition into a commercial-stage gene therapy company within the next two years.
