Inherited Retinal Diseases

In the LIGHTHOUSE study, Atsena Therapeutics is evaluating ATSN-201 gene therapy for X-linked retinoschisis, leveraging AAV.SPR capsid for central retina transduction without foveal detachment risks.

Kaczmarek has a notable background of executive leadership and operational expertise in the gene therapy manufacturing industry.

This interview comes at the Children's Hospital Los Angeles team celebrates its 100th retinal gene therapy procedure.

The aim of this program is to help advance junior clinical research scientists in their professional endeavors to cure retinal degenerative disease.

Data show promising early improvements in low luminance visual acuity, a key measure of visual function.

This virtual key opinion leader (KOL) event will take place on Wednesday, December 11, 2024, at 4:00 PM ET.

Dosing in the first phase of the phase 1/2 OCU410ST GARDian trial for Stargardt disease is complete.

The designations underscore the unmet need in Stargardt disease, a rare and serious pediatric disease for which no treatment exists.

The acquisition creates a clinical-stage company focused on the development of gene therapy treatments for rare inherited retinal degenerations.

Christine N. Kay, MD, discussed her presentation, "Deuterated vitamin A preserves vision in Stargardt’s disease (TEASE study)" which was given at this year's American Academy of Ophthalmology meeting held in Chicago, Illinois.

In an interview with Ophthalmology Times, Quan Dong Nugyen, MD, MSc, discusses ongoing research into Tinlarebant, an investigational drug being studied as a treatment for Stargardt disease in adolescents.

This patented technology is designed to enhance the light sensitivity of a multi-characteristic opsin developed by Nanoscope as a gene therapy.

According to the company, the platform accompanies the launch of Qdata Retinitis Pigmentosa for life sciences and the development of Practice Insights for clinicians.

According to the company, MCO-010 is an ambient-light activatable Multi-Characteristic Opsin (MCO) optogenetic therapy for vision restoration in advanced retinitis pigmentosa (RP), irrespective of gene mutation.

Tinlarebant is an oral therapy designed to reduce the accumulation of vitamin A based toxins that cause retinal disease.

The NIH-supported findings could pave the way for genetic testing, clinical trials, and therapy development for these diseases.

The announcement marks the second FDA designation for ATSN-201, which previously received Rare Pediatric Disease designation.

Retina is a rapidly advancing field with promising research, technology, and treatment areas. Two stand out as particularly exciting.

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.