NAION the Horizon

The exact causes of non-arteritic anterior ischemic optic neuropathy (NAION) are unknown – and no effective treatments exist. What we do know is that NAION occurs more commonly in patients with existing conditions, such as diabetes, decreased blood pressure, and sleep apnea. It has also been noted that smoking and erectile dysfunction medications may increase NAION risk, but these links are not yet proven. Preclinical data from a close translation model in non-human primates implies that a new gene therapy under development by Life Biosciences may have the ability to restore vision in those affected by NAION (1).

“NAION is a sudden onset blinding disease with no treatment options that can be modeled in nonhuman primates (NHPs),” says Sharon Rosenzweig-Lipson, Chief Scientific Officer of Life Biosciences and one of the study’s main authors. “So it was important that we build on the existing data and bridge to NHPs, whose eye structure and function is closely aligned with humans.” Determining whether the model could in fact be transferred to NHPs, the researchers treated NAION-induced African green monkeys with AAV2-OSK (adeno-associated virus type 2 induced genes Oct4, Sox2 and Klf4) and oral doxycycline. The results demonstrate restoration of key aspects of visual function in the NHPs and provide proof-of-concept for the therapy’s potential not only in NAION but also other retinal disorders.

Speaking of the translational implications of the study, Rosenzweig-Lipson says, “The ability of OSK to demonstrate efficacy across both mice and nonhuman primates is extremely encouraging and could be transformative for patients with NAION, who currently have no treatment options.”

Additionally, Rosenzweig-Lipson believes OSK treatment could have wider-reaching implications. “Ophthalmological disorders, including NAION and glaucoma, caused by retinal ganglion cell dysfunction are just the beginning,” she says. “Other age-related diseases of the eye and non-ocular neurodegenerative disorders may be treatable using OSK. Life Biosciences is focused on the tremendous opportunities available to applying OSK technology in a broad range of age-related diseases, and exploring these multiple opportunities as quickly and efficiently as possible.