OCU400 is a novel gene therapy platform with the potential to be broadly effective in restoring retinal integrity and function across a range of genetically diverse inherited degenerative retinal diseases (IRDs). The unique approach leverages therapeutic modifier genes to restore retinal homeostasis and rescue the defects caused by inherited gene mutations, modifying retinal disease progression through the regulation of key gene networks as the site of disease.
Unlike single-gene replacement approaches, which have shown tremendous promise in rare retinal diseases despite being highly specific for a single condition, OCU400 represents a powerful and remarkably broadened means of treating a variety of IRDs with a single therapy. Ocugen is advancing its OCU400 platform in an effort to create vision-sparing therapies for rare/orphan IRDs such as S-cone syndrome, Goldman-Favre syndrome, and retinitis pigmentosa, as well as other forms of retinal and macular degeneration.
Based on cutting-edge technology from the Schepens Eye Research Institute of Massachusetts Eye and Ear, OCU400 involves targeted delivery and expression of one or more nuclear hormone receptor genes (NHRs), which play a vital role in regulating retinal cell development, maturation, metabolism, visual cycle function, and survival. Multiple animal models have shown that expression of NHRs within the retina can stabilize retinal cells and prevent the development of inherited disease. OCU400-based therapies may have the potential to halt disease progression and improve outcomes for people with blinding retinal diseases.
Ocugen and its development partners are currently working to select a lead candidate therapy and delivery method for preclinical and clinical development.
About Inherited Retinal Diseases
Many degenerative retinal conditions are caused by genetic mutations that are passed down within families and lead to progressive disease, severe visual impairment and blindness. Unfortunately, there are a multitude of known mutations in multiple different genes that cause a variety of IRDs. Treating these conditions has been a significant challenge due to the sheer volume of potential therapeutic targets. Gene replacement therapy is a promising approach to providing lasting restoration of normal retinal function, but such therapies can only address one gene at a time, limiting their effectiveness. OCU400 is designed to modify the expression of key networks of genes involved in maintaining retinal homeostasis, potentially compensating for the impairment caused by the original gene mutation.
- Cruz NM, Yuan Y, Leehy BD, Baid R, Kompella U., et. al. (2014)
Modifier Genes as Therapeutics: The Nuclear Hormone Receptor Rev Erb Alpha (Nr1d1) Rescues Nr2e3 Associated Retinal Disease, PLoS ONE 9(1): e87942.