Inherited Retinal Diseases

The submission was based on the safety and efficacy data from the phase 3 (RHODOS) and phase 4 (LEROS) studies.

Belite Bio completes its phase 3 trial for tinlarebant, a potential first treatment for Stargardt disease, with results expected in late 2025.

Kwangdong, a top 5 pharmaceutical and healthcare company in Korea, is actively involved in research and development innovation, including “transformational late-stage, high-impact technologies.”

Researchers will investigate a gene in the eye that is crucial for normal vision, but can cause retinal diseases when mutated that often lead to blindness.

Beacon Therapeutics shares promising phase 2 trial results for laru-zova, a gene therapy showing potential in treating X-linked retinitis pigmentosa.

Alfredo Sadun, MD, PhD, details an unexpected outcome from a gene therapy in development for Leber hereditary optic neuropathy (LHON).

Prof. Androudi said differential diagnoses are more important than ever in the era of cell and gene therapies.

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

jCyte initiates patient dosing in a new trial for famzeretcel, aiming to transform treatment options for retinitis pigmentosa.

Catch up on recent advancements in retina care, including new therapies for age-related macular degeneration and macular telangiectasia, enhancing patient outcomes.

Enhanced screenings and a unified registry could save vision and lives.

NICE approves idebenone for Leber hereditary optic neuropathy, offering hope for vision restoration and improved quality of life for patients.

Opus Genetics partners with the Global RDH12 Alliance to advance gene therapy for inherited retinal disease, aiming for FDA application by late 2025.

Findings from a phase 2 clinical trial support the therapeutic potential of ADX-2191 in patients with retinitis pigmentosa.

The platform, developed by University College London and Heidelberg Engineering, demonstrated accuracy and precision in early tests.

AAVB-039 advances gene therapy for Stargardt disease, aiming for innovative treatments for inherited retinal disorders.

The completion of the full BLA submission is anticipated in early 2026 and is eligible for priority review under the program’s fast-track designation.

RetinalGeniX partners with Labcorp to enhance genetic testing and retinal imaging, aiming for early disease detection and improved patient care.

The VISTA trial is evaluating and comparing 2 dose levels of laru-zova with an untreated control group for the treatment of X-linked retinitis pigmentosa (XLRP).

Atsena Therapeutics expands its ATSN-201 trial for X-linked retinoschisis, a step toward the first gene therapy treatment.

Cell and gene therapies in development for unmet needs.