The Promise of Cell and Gene Therapy for Glaucoma Treatment

According to the National Eye Institute, glaucoma constitutes the foremost cause of blindness and vision loss in the United States. Notably, it has no symptoms early on, and can only be diagnosed through a comprehensive dilated eye exam. Although some treatments for the condition are available, it has no cure, and substantial unmet need remains for the patient population. One area of interest for new glaucoma treatments is cell and gene therapy. This year for Glaucoma Awareness Month, which is observed annually in January by the patient and clinician communities, CGTLive® reached out to Jeffrey Goldberg, MD, PhD, a professor and chair of ophthalmology at the Byers Eye Institute at Stanford University, to learn more about how cell and gene therapy might help to address unmet needs in glaucoma. CGTLive: Can you give some background on unmet needs in glaucoma? Jeffrey Goldberg, MD, PhD: The biggest unmet need is for neuroprotective therapies—to prevent patients from losing their vision—and therapies that can restore vision in those patients who have already lost vision. How could glaucoma potentially be addressed by cell or gene therapy approaches? In early or moderate disease, gene therapy could deliver a neuroprotective or even neuroenhancing therapy that prevents vision loss, or gives a booster shot to sick but not yet dead retinal ganglion cells, thereby protecting or restoring vision. Cell therapy could be used to replace retinal ganglion cells, the neurons that degenerate in glaucoma, and rebuild the optic nerve from the eye to the brain, restoring vision. Are there any ongoing research/development programs for cell/gene therapy in glaucoma that you can discuss? There are many gene therapy programs that have demonstrated strong preclinical efficacy and are moving through the pipeline towards human trials. Cell therapy programs for retinal ganglion cell replacement are a few years further behind—we still have to solve significant problems in the science, including how to optimally deliver the donor cells such that they integrate into the patient retina, and grow their axon fibers down the optic nerve to the brain, to restore that connection and the patient’s vision. The work is moving quickly, but there is still more to be done before this reaches clinical trials. What is your main message to doctors and the broader healthcare community for Glaucoma Awareness Month in 2026? We are on the cusp of seeing the biomedical revolution translate into actual therapies for patients—it is a time of great hope and great excitement! This transcript has been edited for clarity.