Inherited Retinal Diseases

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.

Dr. Gonzalez shares 126-week topline results from the REMAIN study, assessing optogenetic therapy MCO-010 (Nanoscope Therapeutics).

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.

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.

Jennifer Lim, MD, FARVO, FASRS, discusses her ARVO presentation on sickle cell retinopathy, highlighting improvements in retinal surgery techniques and instrumentation.

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.

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