March 20, 2024 — A new gene therapy from Oculogenex Inc., a biotech company, seeks to develop an effective treatment to improve vision or slow disease progression for people with age-related macular degeneration (AMD). An investigation to test the therapy in space can potentially provide new opportunities for treating vision loss.
Experiments have shown Oculogenex’s therapy is effective against retinal damage in models of retinal degeneration, but these models do not emulate intermediate AMD’s milder, chronic oxidative stress. To address this, the International Space Station (ISS) National Laboratory is sponsoring an investigation set to launch of SpaceX’s 30th Commercial Resupply Services mission to ISS. The effort seeks to validate spaceflight as a novel biologic model of intermediate AMD, allowing Oculogenex to evaluate its therapy in a space environment to replicate the hard-to-study stage of the condition, in addition to determining whether gene therapy can thwart spaceflight-induced retinal dysfunction and degeneration, the ISS National Laboratory explains.
“The microgravity investigation could pave the way for future clinical trials and treatments, offering hope to those grappling with this debilitating condition,” says CEO of Oculogenex Hema Ramkumar, M.D., a physician who treats people affected by macular degeneration in California.
Oculogenex shares that its gene therapy enhances cellular response to oxidative stress and prevents retinal cell death, potentially restoring the functionality of damaged cells and preventing vision loss.
“As we age, an epigenetic switch stops cellular repair, leading to cell degradation. Our gene therapy renews dormant retinal stem cells, enhances mitochondrial function, and prevents cell senescence and death,” says Dr. Ramkumar. “By targeting this epigenetic switch, our therapy instructs cells to remain active and resilient, promoting repair and sustaining vision.”
Oculogenex’s gene therapy is designed to target the root causes of dry macular degeneration at the intermediate stage. Because macular degeneration disproportionately affects women, female rodents will be used to conduct the experiment in the space station, with spaceflight-induced oxidative stress serving as a disease model for AMD, mirroring pathways observed in patients on Earth.
“By harnessing the distinctive conditions of spaceflight, we aim to gain invaluable insights into the potential of spaceflight as a model of dry AMD,” says Dr. Ramkumar. “We anticipate our transformative gene therapy strategy will translate into improved and preserved vision for the millions living with dry macular degeneration.”