Inherited Retinal Diseases

Benjamin Bakall, MD, PhD, weighs the RESTORE efficacy and safety data, the practical realities of in-office delivery, and how genotype-independent treatment reshapes candidacy and patient counseling.

The authors of a review of 16 gene therapy trials for inherited retinal diseases explain why the pediatric evidence base remains thin and argue that individual-level, age-stratified reporting is the change most needed before CRISPR-based editing reaches children.

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.

Zero Blind Babies uses wide-field retinal imaging and telemedicine to expand ROP screening across Ghana, Ethiopia, Tanzania, and Syria—screening 400 infants weekly at $56 per QALY—while stressing that local staff and complete-pathway systems remain essential.

Japan designated gildeuretinol an orphan drug for Stargardt disease as the oral therapy enters phase 3 evaluation in the NORTHSTAR trial.

Byron Lam, MD, and Benjamin Bakall, MD, PhD, discuss what an FDA approval of tinlarebant could mean for the Stargardt disease type 1 standard of care.

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.

FDA grants priority review to Belite Bio's tinlarebant NDA for Stargardt disease type 1, setting a February 2027 PDUFA date for a potential first STGD1 therapy.

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

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.

Danon disease is a rare, multisystem X-linked genetic disorder caused by variants in the LAMP2 gene. In children and young adults, it is defined overwhelmingly by its cardiac effects . What is far less appreciated is that the eye is frequently involved and that the retinal findings can appear before the systemic diagnosis is established.

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.

Cynthia Toth, MD, of Duke University, discusses new BabySTEPS research showing that retinal nerve fiber layer and choroidal thickness on OCT correlate with 2-year neurodevelopmental outcomes in preterm infants and what it will take to bring bedside screening into routine NICU care.

How this gene-agnostic optogenetic approach could fill a critical gap for advanced inherited retinal degeneration.

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.

Swiss regulator backs tinlarebant for Stargardt as DRAGON phase 3 shows slower retinal atrophy, bringing first treatment closer to patients.

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.

According to the company, the NDA submission is supported by data from the phase 3 DRAGON trial.

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.

FDA fast-tracks RTx-015, an optogenetic gene therapy aiming to restore vision in late-stage retinitis pigmentosa.

Aflibercept briefly reverses severe ROP in tiny preemies, yet nearly half relapse within weeks—prompting laser rescue and renewed safety questions.

New MLSDT functional vision test tracks advanced retinitis pigmentosa better than acuity, supporting future gene and optogenetic therapy endpoints.

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

Dr. Quan Dong Nguyen presents interim data from the phase 3 DRAGON study on a potential first therapy for adolescent Stargardt disease.