Inherited Retinal Diseases

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.

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.

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.

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.

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.

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.

Combining imaging and patient symptoms improves assessment of disease progression.

Dr. J. Peter Campbell discusses the technologies shaping retinopathy of prematurity detection, clinical integration, and global implementation.

According to the company, this is the first and only single-use kit on the market that includes the diagnostic lens.

Both therapeutics will leverage AGC Biologics’ BravoAAV suspension platform and use an innovative dual AAV vector approach, which splits the therapeutic gene into 2 halves.

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).

Best disease, also known as vitelliform macular dystrophy, is a rare, inherited retinal condition causing macular degeneration by mutations in the BEST1 gene, leading to progressive vision loss and potentially blindness.

Kenneth C. Fan, MD, discusses promising visual improvements from MCO-010 therapy for Stargardt disease at AAO 2025, highlighting future treatment potential.