MeiraGTx Holdings has reacquired full rights to botaretigene sparoparvovec (bota-vec) for the treatment of X-linked retinitis pigmentosa (XLRP) from Johnson & Johnson, the company announced. The agreement includes a $25 million upfront payment and future milestone and royalty obligations.

Bota-vec is an adeno-associated viral vector gene therapy targeting RPGR mutations, a common cause of XLRP. The condition leads to progressive vision loss, often resulting in blindness by early adulthood, and currently has no approved treatments.

The phase 3 LUMEOS trial (n=95) did not meet its primary endpoint, a visual mobility assessment, although treated patients were more likely to respond than controls. Secondary endpoints showed statistically significant improvements across multiple measures, including retinal sensitivity and low-luminance visual acuity (LLVA). Approximately 45% of treated patients gained at least 10 letters in LLVA, and 20% gained 15 letters or more.

Patient-reported outcomes also favored treatment, with improvements in mobility and dim-light function. Safety findings were consistent with earlier studies, with no new signals reported.

MeiraGTx said it plans to file biologics license and marketing authorization applications in the United States, European Union, and Japan. The company indicated that manufacturing validation is complete and that commercial supply is available, with a potential launch targeted for 2027.

The US Food and Drug Administration (FDA) has granted Fast Track and Orphan Drug Designations to bota-vec, and regulatory authorities in the European Union have granted Priority Medicines (PRIME), advanced therapy medicinal product (ATMP), and Orphan Drug Designations to bota-vec.