Gene Therapy

Opus Genetics announced positive 3- and 6-month results OPGx-BEST1 in BEST1-related retinal diseases.

The FDA accepted Nanoscope's BLA for MOGENRY (sonpiretigene isteparvovec, MC-010), a gene therapy for retinitis pigmentosa with severe vision loss, supported by RESTORE trial data showing sustained BCVA improvement.

Jay Chhablani, MD, on how MacTel eyes turn neovascular, when to refer, and what Encelto means for treatment.

João Pedro Marques, MD, PhD, on how systematic reanalysis moved a cohort's solved rate from 59.4% to 70.1% and what it means for gene therapy eligibility

Noah G. Banoub discusses a study finding MacTel2 misdiagnosed in 25.5% of cases, common mimics, misleading imaging, and Encelto's approval.

Patient enrollment is complete in a phase 3 registrational, or pivotal, trial to evaluate OPGx-LCA5, a gene therapy that is under investigation for treating Leber congenital amaurosis type 5-associated inherited retinal disease.

Ahead of his November 10, 2026, start date, the incoming Duke ophthalmology chair shares his vision for research, training, and the integration of AI and data science.

An investigational treatment for vision loss associated with Bardet-Biedl syndrome is being tested in the first three patients treated.

The FDA has cleared an IND for RAG-1C, a first-in-class saRNA therapy targeting proliferative vitreoretinopathy.

Charles Wykoff, MD, PhD, of Retina Consultants of Texas, discusses 1 year of real-world revakinagene taroretcel experience, covering patient selection, surgical technique, early safety outcomes, and the platform’s potential beyond MacTel.

Rare retinal diseases initially were the primary targets of gene therapy, but that is no longer the case. The focus has expanded beyond those to also include more common, chronic diseases such as diabetic macular edema and wet age-related macular degeneration. During the first half of 2026, that trend was evident.

A successful Type B Rare Disease Evidence Principles meeting confirms the pivotal study design, opening a potential BLA pathway on 6-month efficacy data, with dosing expected to begin in the fourth quarter of 2026.

Three-year gene therapy data, an FDA reversal in wet AMD, light-powered dry eye treatment, a new macular supplement, and progress in childhood glaucoma—our review breaks down the 5 May 2026 stories that matter.

Alkeus Pharmaceuticals has dosed the first participant in the phase 3 NORTHSTAR trial of oral gildeuretinol for Stargardt disease, a condition with no approved treatments.

Guidance includes building on existing science, reducing redundant testing, and accelerating treatments for rare and life-threatening diseases

Two leading retinal specialists break down MCO-010's 3-year efficacy and safety data, the significance of contralateral eye findings, and how this gene-agnostic optogenetic approach could fill a critical gap for patients with advanced inherited retinal degeneration who have limited treatment options.

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.