Inherited Retinal Diseases

Michel Michaelides, MD, discusses phase 3 DRAGON results showing that oral tinlarebant slowed atrophic lesion growth by about 36% in adolescents with Stargardt disease.

Three researchers share the prize for discovering channelrhodopsin, a light-sensitive protein now being tested to restore vision in retinal disease.

Ester Carreño, MD, PhD, explains how to tell retinitis pigmentosa from uveitis and why genetic confirmation now matters more.

Japan's filing covers IRDs broadly, supported by RESTORE and open-label Stargardt data.

Inherited metabolic diseases affect the retina even before the systemic diagnosis is established.

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.

Topline data from the phase 2/3 study show that both dose levels of the subretinal gene therapy produced significant gains in low luminance visual acuity versus untreated control at 12 months.

The FDA has accepted Nanoscope's BLA for sonpiretigene isteparvovec in retinitis pigmentosa, backed by RESTORE and REMAIN trial data.

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.