Inherited Retinal Diseases

According to research conducted by investigators at the Perelman School of Medicine at the University of Pennsylvania School of Medicine, patients with a rare inherited disease affecting their sight experienced quick vision improvements that sustained for the full year-long study.

The one-time intravitreally delivered gene therapy is for the treatment of retinitis pigmentosa (RP).

The 4-year study is set to involve up to 20 patients and is a part of the Foundation's Uni-Rare natural history study.

The product candidate is a modifier gene therapy for broad retinitis pigmentosa indication.

According to the company, the priority review voucher would be issued upon approval of OPGx-LCA5.

The LIGHTHOUSE study a Phase I/II, open-label, dose-escalation and dose-expansion clinical trial is currently investigating the safety and tolerability of ASTN-201 for XLRS.

Tim Cherry, PhD, and Debarshi Mustafi, MD, PhD, have been awarded a $150,000 Clinical & Research Synergy Pilot Award from Seattle Children’s Research Institute to determine the genetic causes of unsolved inherited retinal diseases.

This clinical milestone marks a new step in treating the condition with a potential long-term benefit.

Researchers at National Taiwan University Hospital concluded that significant differences were observed in visual acuity, refractive state and myopia rate between patients with inherited retinal disease and the general population.

KIO-30 is a small molecule photoswitch that selectively confers light-sensing capabilities to retinal ganglion cells following the degeneration of photoreceptors in inherited retinal diseases.

Stephen Russell, MD, discussed his presentation on safety and durability of voretigene neparvovec-rzyl for biallelic RPE65 at the annual ASRS meeting in Stockholm, Sweden.

Richard B. Rosen, MD, from the New York Eye and Ear Infirmary of Mount Sinai discussed his presentation on sickle cell retinopathy and the measurement with dynamic OCT angiography and quad-fusion adaptive optics imaging at the annual ASRS meeting in Stockholm, Sweden.

Michael Singer, MD, presented 100-week results from the RESTORE trial and shares key takeaways from his presentation at this year's American Society of Retina Specialists (ASRS) meeting.

Data in early-stage patients was presented at 42nd American Society of Retina Specialists Annual Scientific Meeting, being held in Stockholm, Sweden.

Patrick C. Staropoli, MD, talked about his presentation on the clinical characterization of Hexokinase 1 (HK1) mutations causing autosomal dominant pericentral retinitis pigmentosa at the annual ASRS meeting in Stockholm, Sweden.

Sydney M. Crago, Assistant Managing Editor of Ophthalmology Times and Modern Retina, spoke with attendees, who shared which topics they are most looking forward to discussing at the ASRS meeting held in Stockholm, Sweden.

Researchers, led by Shigemi Matsuyama, PhD, will conduct research seeking a potential breakthrough drug that can be taken orally—one that may address many RP disease manifestations, regardless of the underlying genetic mutation.

AAV204 is a novel adeno-associated virus (AAV) capsid from the AIM capsid library licensed by Abeona from the University of North Carolina at Chapel Hill.

The organization has secured £72.75 million for its new eye health center.

We asked what leaders in the retina field would tell residents and medical students as they train for the next decades in retinal care. Here's what 2 of them had to say.

OCU410ST is a modifier gene therapy candidate being developed for Stargardt disease, which affects approximately 100,000 people in the United States and Europe combined.

OCU400 is a gene-agnostic modifier gene therapy product based on nuclear hormone receptor (NHR) gene, NR2E3.

VISTA is a global randomly assigned, controlled, masked, multi-center pivotal study evaluating the efficacy, safety, and tolerability of 2 dose levels of AGTC-501 for the treatment of X-linked retinitis pigmentosa

The trial evaluates OCU410ST, a modifier gene therapy candidate utilizing an AAV delivery platform for the retinal delivery of the RORA gene.

Researchers at Massachusetts Eye and Ear led a phase 1/2 trial, which included 14 participants, that found the experimental treatment was safe and efficacious.

Christine Kay, MD, spoke with Modern Retina to share insights from her presentation on longitudinal BCVA and safety analysis of mutation-agnostic MCO-010 optogenetic therapy For retinitis pigmentosa

The data will be presented in oral session at the 2024 American Society of Gene & Cell Therapy meeting May 7-11 in Baltimore, Maryland.

This initiative will provide a variety of tools to promote awareness and education, including some resources in both English and Spanish.

The Vision Simulator shows disease progression and vision loss simulations for diabetic macular edema, diabetic retinopathy, AMD, XLRP, and achromatopsia.

According to the company, SGT-1001 uses its novel gene coding technology for gene integration via transposition. It plans to advance SGT-1001 into the clinic in the first half of 2025. Preclinical data for SGT-1001 will be presented at the Association for Research in Vision and Ophthalmology and the American Society of Gene & Cell Therapy annual meetings.