Inherited Retinal Diseases

MCO-010 receives Orphan and RMAT designations, highlighting its potential as a groundbreaking therapy for various retinal dystrophies.

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.

Health Canada approves Aflivu, a biosimilar for retinal diseases, enhancing treatment options and affordability for Canadian patients.

Cell and gene therapies in development for unmet needs.

Nanoscope Therapeutics establishes a Vision Advisory Committee to enhance retinal care and drive innovative therapies for vision restoration.

Phase 1/2 interim results highlight real-world functional enhancements in patients with severe LCA5-related Leber congenital amaurosis.

PYC Therapeutics advances VP-001 for retinitis pigmentosa, securing FDA guidance for its upcoming registrational trial design and study parameters.

The phase 2/3 clinical trial for OCU410ST will enroll 51 participants diagnosed with Stargardt disease.

A mutation-agnostic gene therapy approach offers new possibilities for patients with advanced retinal disease.

In a recent study, the researchers suggested that targeting the circuits responsible for visual acuity may be necessary in achieving optimal recovery of visual function and could aid in the development of vision restoration therapies.

OCU400 is Ocugen’s novel modifier gene therapy for retinitis pigmentosa (RP).

VG801 is a dual AAV gene therapy that leverages mRNA trans-splicing via the vgRNA REVeRT and vgAAV platforms to deliver the full-length ABCA4 gene for Stargardt disease and related retinal disorders.

Retina specialist Jay Chhablani, MD, discusses 3D choroidal vessel segmentation, uncovering critical insights into macular degeneration, diabetic retinopathy, and potential systemic disease connections using advanced OCT technology.

ABCA4-associated retinopathies include Stargardt disease, retinitis pigmentosa 19, and cone-rod dystrophy 3.