Inherited Retinal Diseases

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.

OPGx-LCA5 is designed to address a form of Leber congenital amaurosis that results from biallelic mutations in the LCA5 gene, which encodes the lebercilin protein, the investigators explained.

PolyActiva and RareSight collaborate to create innovative therapies for rare pediatric retinal diseases, aiming to transform treatment and improve children's vision.

Stephen Tsang, MD, PhD, reviews promising optogenetic therapy results for retinitis pigmentosa and Stargardt disease, enhancing vision and mobility for patients.

The EXTEND study is a 5-year follow-up of participants who received a single intravitreal injection of MCO-010 in a previously conducted phase 1/2a trial.

The funding will support the completion of the clinical PoC of its AAVB-039 CELESTE study and the completion of the STELLA natural history study.

A mutation-agnostic gene therapy approach offers new possibilities for patients, especially those with inherited retinal diseases.

NYRVANA is the first-in-human trial and is an open-label, multicenter, dose-escalation study investigating the safety, tolerability, and preliminary efficacy of a single intravitreal injection of SPVN20 over 6 months.

A new study reveals a promising drug reduces vision loss in Stargardt disease, showing significant safety and efficacy over 2 years.

The FDA describes the RDEA pilot program as a program to support novel endpoint efficacy development for drugs that treat rare diseases.

A recent study uncovers a novel biomarker for retinal vascular diseases, highlighting the significance of intermittent capillary perfusion in monitoring treatment efficacy.

Additionally, the company also received Clinical Trial Authorization (CTA) approval from the UK's Medicines and Healthcare Products Regulatory Agency (MHRA).

NPI-001 is Nacuity’s investigational therapy for the treatment of patients with retinitis pigmentosa (RP).

The trial results show promising for the evaluated gene therapy for the treatments of LCA, showing significant vision improvements in pediatric patients.