Inherited Retinal Diseases

Study finds that 1 in 3 children with sickle cell disease has retinopathy.

The company presented topline Phase I/II first-in-human results at the 127th AAO annual meeting in San Francisco, demonstrating significant improvement in visual field, in concordance with trended improvements in visual acuity and functional vision.

Researchers have delved into the possibility of cell-based therapy in ophthalmology. By targeting vitreoretinal diseases, they are tackling a range of vision-threatening disorders which often result in severe irreversible vision loss.

Researchers note their work reports a novel candidate gene for FEVR, which expands the spectrum of EMC1 variants and EMC1-related phenotypes, providing evidence for the prenatal diagnosis of candidate disease-causing EMC1 variants in FEVR.

The research will focus on the compound's impact on biomolecular condensates, which are implicated as a key driver of pathology in neurodegeneration and diseases impacting high metabolic organs. Because the retina is one of the highest energy consuming systems in the human body, deficits in energy supply can be catastrophic.

With a robust pipeline, new hope for Stargardt disease and retinitis pigmentosa may be on the horizon.

The use of VR mazes hold an intrinsic advantage compared with physical mazes in the ability to control conditions and test various patterns with reliability, participant safety, and repeatability.

According to the company, OPGx-LCA5 is designed to address vision loss due to Leber congenital amaurosis associated with mutations in the LCA5 gene, which causes one of the most severe early-onset retinal dystrophies.

According to the companies, the collaboration has the potential to benefit patients who have suffered from specific, chronic ocular diseases.