MeiraGTx Aims for Accelerated Approval of Bota-vec in 2027 Following Reacquisition from Johnson & Johnson

On April 16 local time, Johnson & Johnson announced its ophthalmic gene therapy botaretigene sparoparvovec.（bota-vec）All rights were returned to the original partner, MeiraGTx, which...$25 million upfront paymentRepurchase. Johnson & Johnson is also eligible for a one-time milestone payment related to the potential approval and commercial performance of bota-vec, as well as future sales royalties.

Bota-vec isA one-time gene replacement therapy based on rAAV5,Aimed at delivering function to the retinaSexRPGR Gene Therapy for X-Linked Retinitis Pigmentosa（XLRP）. This is a rare hereditary eye disease that primarily affects men, and patients typically become legally blind around the age of 40.

During the Christmas period in 2023, Johnson & Johnson, through its Janssen division, for up to$415 millionJohnson & Johnson acquired all rights to bota-vec through a full transaction. However, in the pivotal Phase III trial announced in May 2025, bota-vec failed to improve patients' visually guided mobility, leading to the trial's failure. This outcome ultimately prompted Johnson & Johnson to decide to divest this asset.

Despite Phase III Study Not Meeting Primary Endpoint, MeiraGTx Remains Optimistic About Bota-Vec’s Regulatory Outlook. The company stated that it has received positive feedback from several doctors involved in the trial, who believe that bota-vec has benefited a large number of patients."Clinically Meaningful Benefit"MeiraGTx plans to seek expedited approval pathways in the United States and the European Union, and if approved,Expected to be launched in 2027。

However, the market is skeptical about this prospect. Affected by this news, MeiraGTx's stock price fell 16% during early trading on April 16. Additionally, bota-vec’s regulatory path may face challenges — under the leadership of the outgoing head of biologics, Vinay Prasad, the FDA approval process for other gene therapies has already been struggling, and with no successor yet appointed, the direction of approval policies remains unclear.

MeiraGTx, headquartered in New York, has the ultimate goal of becoming a commercialized enterprise. It plans to promote bota-vec and another treatment for radiation-induced xerostomia within the next two years.Gene Therapy AAV-hAQP1 Launched。

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