J&J returns eye disease gene therapy to MeiraGTx

MeiraGTx Holdings has entered into an asset purchase agreement with Johnson & Johnson to reacquire all interests in bota-vec, an investigational AAV5 gene therapy for the treatment of X-linked retinitis pigmentosa (XLRP), a rare genetic eye disease.

J&J’s Janssen unit had purchased the remaining interests in bota-vec from MeiraGTx in December 2023 for $130 million upfront. In May 2025, the therapy failed to meet its primary endpoint — vision-guided mobility — in a phase 3 XLRP trial. However, all but one of the secondary endpoints, including measures from all three visual domains – functional vision, retinal function and visual function – showed benefit.

Now, MeiraGTx will pay J&J a $25 million upfront cash payment and a one-time regulatory and commercial milestone tied to U.S. approval and U.S. sales performance of bota-vec for the treatment of XLRP, as well as a high double-digit royalty on global net sales starting in mid-2029.

In reacquiring bota-vec, MeiraGTx says it intends to “expeditiously” file for approval in the U.S. and EU with the aim of a potential launch in 2027.