Gene Therapy

Data presented at ARVO 2026 show that high-dose gene therapy boosts central retinal sensitivity in X-linked retinitis pigmentosa, with the phase 3 VISTA study fully enrolled.

Interim 6-month results from Part B of the LIGHTHOUSE trial show structural and functional improvements consistent with earlier cohorts; pivotal Phase 3 enrollment now underway with BLA filing targeted for 2028.

A closer look at the science powering next-generation retinal treatments.

The ongoing GARDian3 study is designed to assess whether a single subretinal administration of OCU410ST can slow structural disease progression in patients with ABCA4-associated retinopathy.

Q1 2026 eye-care roundup: first dual-agent presbyopia drop, new EDOF IOL, wet AMD phase 3 win, and gene-therapy advances.Major ophthalmic events in the first quarter of 2026 included treatments for presbyopia, age-related macular degeneration (AMD), dry eye, glaucoma, corneal dystrophy, geographic atrophy, and a new intraocular lens (IOL), among others.1 Key FDA approvals & regulatory actions during Q1 2026) Yuvezzi 2.75%/0.1% (carbachol/brimonidine, Tenpoint Therapeutics Ltd.) was approved as the first dual-agent eye drop designed to treat presbyopia in adults. The dual actions include improving the near vision without decreasing the distance vision.2 TECNIS PureSee intraocular lens (IOL). On March 12, 2026, Johnson & Johnson announced that the FDA approved the TECNIS PureSee extended-depth-of-focus IOL for cataract surgery.3 The launch is planned for later in the year. A full 97% of patients implanted with the IOL reported having no “very bothersome visual disturbances,” according to the company. GEB-101: GenEditBio received Investigational New Drug (IND) clearance to start an in vivo gene-editing trial targeting TGFBI corneal dystrophy in January 2026. This allowed the initiation of the phase 1/2 CLARITY trial to evaluate the safety and efficacy of the CRISPR-based gene therapy for corneal dystrophy associated with the TGFB1 gene.4 OPGx-BEST1: Opus Genetics reported positive initial phase 1/2 data for OPGx-BEST1 to treat Best vitelliform macular dystrophy and autosomal recessive Bestrophinopathy on February 27, 2026. The data showed that the first patient tolerated the drug well with no serious adverse effects. The 3-month results showed a 12-letter increase in visual acuity and decreased central subfield thickness.5 Axpaxli/Duravyu (axitinib/vorolanib). Ocular Therapeutix reported topline data from the phase 3 SOL-1 trial of Axpaxli/Duravyu (axitinib/vorolanib) for wet age-related macular degeneration (AMD) on February 17, 2026.6 The study met its primary endpoint, with 74.1% of patients treated with Axpaxli maintaining vision at week 36 compared to 65.9% in the control (aflibercept, Eylea, Regeneron Pharmaceuticals) group, according to the report. Axpaxli showed superiority over a 2-mg dose of aflibercept, though the magnitude of the benefit was lower than some investors expected, according to eyewire. Geographic atrophy (GA). Ocugen Inc. released its phase 2 data for OCU410 (AAV5-RORA), a subretinal gene therapy for GA secondary to dry AMD, on January 15, 2026.7 The data showed positive preliminary results at the 12-month timepoint. The key results, according to the company, were a 46% decrease in lesion growth (medium + high dose) compared to the control, with a 50% responder rate, demonstrating significant potential for treating this. In addition, the full data set was released on March 24. The key takeaways were as follows: Medium-dose OCU410 met the 12-month primary endpoint, reducing GA lesion growth by 31% in baseline lesions 2-17.5mm², and by 33% in subgroup lesions 5-17.5mm². Structural biomarkers aligned with disease-modifying activity, including 27% slower ellipsoid zone loss and 55% of treated patients achieving ≥30% lesion-growth reduction relative to control. Pipeline Update Reproxalap (Aldeyra Therapeutics) received a third complete response letter for reproxalap.8 The FDA concluded that the New Drug Application lacked substantial evidence of efficacy for dry eye signs and symptoms despite no safety/manufacturing deficiencies. Triesence (Harrow) received IND clearance for a phase 3 trial. The drug is used to better visualize the vitreous intraoperatively. NTX-1024 (NexEos Bio) obtained Orphan Drug Designation for NTX-1024 to treat a protein involved in vernal keratoconjunctivitis. References Charters L. Ophthalmology pipeline watch: Key trial results and PDUFA dates for Q1 2026. Ophthalmology Times.https://www.ophthalmologytimes.com/view/ophthalmology-pipeline-watch-key-trial-results-and-pdufa-dates-for-q1-2026#:~:text=Key%20Takeaways,a%20particular%20focus%20of%20ophthalmologists. Ernst D. Presbyopia. Ophthalmology Advisor. https://www.ophthalmologyadvisor.com/news/yuvezzi-approved-as-once-daily-combination-therapy-for-presbyopia/#:~:text=The%20most%20common%20adverse%20reactions,the%20second%20quarter%20of%202026.&text=References:,.yuvezzi.com/pi. Johnson & Johnson. https://www.jnj.com/media-center/press-releases/johnson-johnson-announces-fda-approval-of-tecnis-puresee-intraocular-lens-a-breakthrough-solution-for-u-s-cataract-patients FDA Clears IND for GenEditBio’s GEB-101 in TGFBI corneal dystrophy. Ophthalmology Management.https://ophthalmologymanagement.com/news/2026/fda-clears-ind-for-geneditbios-geb101-in-tgfbi-corneal-dystrophy#:~:text=FDA%20Clears%20IND%20for%20GenEditBio's,and%20deposits%20in%20the%20cornea. Opus Genetics.Announces Initial Clinical Data from Phase 1/2 OPGx-BEST1 Gene Therapy Study at the Macula Society Annual Meeting. https://ir.opusgtx.com/press-releases/detail/520/opus-genetics-announces-initial-clinical-data-from-phase-12-opgx-best1-gene-therapy-study-at-the-macula-society-annual-meeting#:~:text=Opus%20Genetics%20Announces%20Initial%20Clinical,%2C%20in%20San%20Diego%2C%20California. Ocular Therapeutix Announces Positive Phase 3 SOL-1 Results Demonstrating Superiority of Axpaxli in Wet AMD. https://eyewire.news/news/ocular-therapeutix-announces-positive-phase-3-sol-1-results-demonstrating-superiority-of-axpaxli-in-wet-amd?c4src=article:infinite-scroll Ocugen announces positive preliminary phase 2 data from ocu410 modifier gene therapy for geographic atrophy secondary to dry age-related macular degeneration. https://ir.ocugen.com/news-releases/news-release-details/ocugen-announces-positive-preliminary-phase-2-data-ocu410/#:~:text=Phase%202%20data%20from%20the%20ArMaDa%20clinical,Phase%201%20and%20Phase%202%20clinical%20trials. Harp MD. Aldeyra receives third complete response letter for reproxalap. Ophthalmology Times. March 17, 2026. https://www.ophthalmologytimes.com/view/aldeyra-receives-third-complete-response-letter-from-the-fda-for-reproxalap-and-the-treatment-of-dry-eye-disease

Chiesi Global Rare Diseases said the agency declined to approve its application and requested additional information for the proposed treatment of Leber hereditary optic neuropathy.

Gene and cell therapies plus AI imaging tools accelerate breakthroughs for rare retinal diseases, spotlighting new trials for RP and Stargardt patients.

Trial shows AAV8 PDE6A gene therapy doesn’t improve retinitis pigmentosa vision in a year and may cause retinal thinning and VA loss.

The trial is evaluating OPGx-MERTK gene therapy for MERTK-related retinitis pigmentosa (RP).

Japan fast-tracks MCO-010 optogenetic eye injection as studies show lasting safety and meaningful vision gains in inherited retinal disease.

The trial is evaluating OCU410 (AAV5-RORA) for the treatment of geographic atrophy (GA) secondary to dry age-related macular degeneration.

OCU410ST uses an adeno-associated virus delivery platform for the retinal delivery of the RORA gene.

The Part B dose-expansion portion is evaluating SB-007 for the treatment of Stargardt disease.

Explore the challenges and insights in long-term follow-up of paediatric gene therapy, focusing on data integrity, patient retention, and ethical considerations.

CoTx-101 is intended for the treatment of retinal vascular diseases, such as wet age-related macular degeneration (wAMD) and diabetic macular edema (DME).

RUSH2A is an ongoing natural history study for patients with mutations in the USH2A gene causing Usher syndrome type 2A or nonsyndromic retinitis pigmentosa (RP).

The EXTEND trial provided 5-year follow-up for 10 patients with advanced retinitis pigmentosa, who had previously received MCO-010 during a phase 1/2a trial

This acquisition will include Adverum’s lead candidate, Ixo-vec, a gene therapy treatment for wet AMD.

SPVN20 is a gene-agnostic, intravitreal AAV-based gene therapy that targets dormant cones to restore vision, according to SparingVision

A mutation-agnostic gene therapy approach offers new possibilities for patients, especially those with inherited retinal diseases

AMD treatment may become unrecognizable as gene therapies evolve.

According to the company, FALCON aimed to provide “a better understanding of how autosomal dominant optic atrophy (ADOA) disease parameters change over time to inform potential future interventional clinical trials.”

As ophthalmic technologies move at supersonic speed, AI and gene therapy take center stage.