March 26, 2026 —
MeiraGTx is continuing to advance a growing pipeline of AAV-based gene therapies, with several programs approaching late-stage development across inherited retinal diseases and other indications. During a recent presentation at an RBC Capital Markets ophthalmology conference, company CEO Alexandria “Zandy” Forbes highlighted the progress of three late-stage clinical programs, including two targeting inherited retinal diseases (IRDs) and another focused on radiation-induced xerostomia.
A central aspect of MeiraGTx’s development strategy is the use of small, localized doses of adeno-associated virus (AAV) vectors, particularly in ophthalmic applications. According to the company, delivering AAV vectors locally rather than systemically may help reduce safety risks and lower manufacturing costs, while still enabling effective gene delivery to targeted tissues.
Inherited retinal diseases are particularly well suited for AAV gene therapy approaches, as many are caused by single-gene mutations that can potentially be corrected through gene replacement strategies. In addition, the eye allows for localized delivery and direct monitoring of treatment effects using established clinical endpoints that measure retinal function and visual performance.
One of MeiraGTx’s most advanced ophthalmology programs targets X-linked retinitis pigmentosa (XLRP) caused by mutations in the RPGR gene. This program is partnered with Johnson & Johnson, which owns the asset, while MeiraGTx serves as the commercial manufacturing partner. According to Forbes, pivotal clinical data presented previously showed “extremely promising” results, with investigators reporting meaningful benefits for patients. The Phase 3 development program involved approximately 30 clinical sites across the United States and Europe, with more than 60 surgeons trained to deliver the therapy. The company is currently awaiting Johnson & Johnson’s strategy for advancing the therapy toward patient access.
Another key program targets Leber congenital amaurosis type 4 (LCA4) caused by mutations in the AIPL1 gene, an ultra-rare inherited retinal disorder that leads to severe visual impairment or blindness in infancy. This AAV gene therapy program has been licensed to Eli Lilly in a deal that included $75 million upfront and up to $135 million in milestones. Under a U.K. “specials” regulatory pathway, which allows physicians to administer investigational therapies in certain cases, 11 infants treated with the AAV-based therapy reportedly experienced restoration of sight, representing a remarkable early outcome for this ultra-rare disease.
MeiraGTx’s work in inherited retinal diseases is supported by long-standing collaborations with University College London and Moorfields Eye Hospital, two globally recognized centers for retinal research and treatment. These partnerships provide access to specialized clinical expertise, patient identification networks, and genotype-driven patient selection.
Beyond inherited retinal diseases, the company is also developing an AAV gene therapy program for radiation-induced xerostomia, a severe and chronic form of dry mouth that can occur after radiation therapy for head and neck cancers. This program is currently in a pivotal Phase II study, and the U.S. Food and Drug Administration has agreed that the trial could potentially support a Biologics License Application (BLA) as a single study. MeiraGTx expects to submit a regulatory filing next year, with a potential commercial launch targeted for 2028.
In addition to its current programs, the company is expanding its gene therapy technology platform, including development of intravitreal AAV capsids and cell-specific promoters designed to enhance retinal targeting. These technologies could enable gene therapy treatments for larger ophthalmic indications such as wet age-related macular degeneration, dry AMD, and glaucoma.
MeiraGTx is also developing a riboswitch-based gene regulation platform, which combines AAV-delivered DNA templates with orally administered small-molecule activators to control protein expression after treatment. The company is preparing an Investigational New Drug (IND) application for a leptin-based therapy using this platform and has reported encouraging preclinical data in additional indications, including neuropathic pain.
Together, these programs highlight the expanding role of AAV gene therapy across multiple therapeutic areas, from rare inherited retinal disorders to broader disease indications where targeted, durable gene expression could transform long-term treatment strategies.
