Khaberni - Life Biosciences, an American biotechnology company, begins clinical trials on the gene therapy ER-100 aimed at restoring vision through "renewing" damaged cells in the retina.
The drug, developed by scientists, is designated to treat visual neurodegenerative disorders that lead to the death of retinal ganglion cells, which are responsible for transmitting visual signals from the eye to the brain. Diseases such as glaucoma or non-arteritic ischemic optic neuropathy can lead to total vision loss.
The treatment relies on genetic reprogramming of cells, by affecting the chemical markers that regulate gene activity without altering the DNA. This is done using three "Yamanaka factors": OCT-4, SOX-2, and KLF-4 (OSK), which play a central role in activating cellular renewal mechanisms. The drug will be administered through an injection into the vitreous body of the eye.
During pre-clinical tests, the treatment showed its ability to restore "youthful markers" in the DNA and improve vision in animal trials, which led the U.S. Food and Drug Administration (FDA) to grant permission to begin trials on humans.
