Gene Therapy

An industry symposium on intravitreal 4D-150 and a course on subretinal delivery lead gene therapy content at EURETINA 2026, alongside IRD courses and trial readouts.

OT Roundup debuts with new ADHD ocular findings, a surgeon's own trifocal IOL journey, MOGENRY's FDA filing, and myopia policy news.

Q3 2026 delivered advances in some closely watched pipeline products.

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

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.

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.

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.

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.