Inherited Retinal Diseases

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.

The system was taught quality standards by being fed more than 1400 images from different parts of the retina taken by adaptive-optics ophthalmoscopy.

The recent study details the diagnostic value of a multimodal model utilizing both types of biomarkers when compared to unimodal models.

Alfredo Sadun, MD, PhD, details a fascinating outcome from a gene therapy in development for Leber hereditary optic neuropathy.

Beacon Therapeutics reveals promising interim results for laru-zova, a gene therapy showing potential to improve vision in XLRP patients.

Opus Genetics reveals promising 1-year results from OPGx-LCA5 gene therapy, showing sustained vision improvements in adults with LCA5 retinal degeneration.

The LUMEOS phase 3 clinical trial resulted in botaretigene sparoparvovec not meeting primary endpoints to improve visual navigation of XLRP.

Prevent Blindness has declared May the second annual inherited retinal disease and genetic testing month, offering free resources on education and other awareness efforts.

Cencora will serve as the exclusive specialty distributor and provide comprehensive commercialization support, including third-party logistics, specialty logistics and patient support services

Administered via intravitreal injection, Synthopsin-MCO-010 possesses a promoter to specifically target ON-bipolar cells.

SpliceBio continues to actively enroll patients in the ASTRA study and accompanying POLARIS trial

ENCELTO is the first and only FDA-approved treatment for MacTel.

The modifier gene therapies from Ocugen target geographic atrophy and Stargardt disease.

Researchers indicated that pediatric patients with Leber congenital amaurosis experienced improvements in vision.

Researchers from the University of Bristol recorded increased instances of inflammation in particular demographic groups

The trial will feature the company’s product candidate, AXV101, focused on combating childhood blindness due to retinitis pigmentosa (RP) caused by Bardet-Biedl Syndrome 1 (BBS1).

Laru-zova is a gene therapy currently being investigated for the treatment of patients with X-linked retinitis pigmentosa.

Previous research has shown that voretigene neparvovec administered subretinally early in childhood for RPE65-mediated inherited retinal dystrophy achieved encouraging efficacious results.

The NPI-001 (N-acetylcysteine amide) tablets are a proprietary investigational therapy for the treatment of patients with retinitis pigmentosa.

OCU400 demonstrated meaningful improvement of 2-line gain (10 letters on ETDRS chart) in low-luminance visual acuity (LLVA) in treated eyes when compared to untreated fellow eyes.

ViGeneron also received approval for dose escalation in the ongoing phase 1b clinical trial.

LHON, a genetic disease that affects the retinal ganglion cells, results in severe bilateral sequential vision loss.

The company is advancing its Phase I/II trial and exploring accelerated approval pathways in the US and Europe.

USC is leading a research team aiming to better understand retinitis pigmentosa and inform future treatments.