MeiraGTx received FDA Breakthrough Therapy Designation for AAV2-hAQP1 and reported positive three-year data from its Phase 1 AQUAx trial involving 24 patients with grade 2/3 radiation-induced xerostomia. Results showed sustained, clinically meaningful improvements in patient-reported outcomes and objective salivary flow measures from 12 through 36 months post-treatment, with no new safety signals.

In April 2026, MeiraGTx entered an asset purchase agreement with Johnson & Johnson to acquire full rights to botaretigene sparoparvovec (bota-vec) for X-linked retinitis pigmentosa. The company paid a $25 million upfront cash consideration and agreed to potential regulatory and commercial milestones plus a mid-teens royalty on global net sales, targeting U.S., EU, UK and Japan filings.

MeiraGTx strengthened its balance sheet by securing $100 million in financing during the first quarter of 2026. The funds are expected to support regulatory submissions, manufacturing scale-up and potential launches of its two lead gene therapy candidates over the next two years.

Penny Fleck joined MeiraGTx as Chief Development Officer, bringing over 20 years of regulatory and development experience from her roles at Johnson & Johnson and Takeda. Stuart Naylor transitioned to Chief Scientific Officer, Ophthalmology, focusing on global regulatory approval of bota-vec and advancement of ophthalmology programs including AAV-RPGR.