ISS National Lab-sponsored study to test a novel gene therapy for AMD


An investigation is set to launch on SpaceX’s 30th Commercial Resupply Services mission to the International Space Station contracted by NASA to test the therapy in space.

Image credit: AdobeStock/3dsculptor

(Image credit: AdobeStock/3dsculptor)

Oculogenex Inc. is poised to shift the paradigm in the treatment of age-related macular degeneration with a gene therapy that may prevent or even reverse symptoms of this condition.

AMD is a leading cause of blindness in older adults, affecting more than 200 million people globally. Patients in the intermediate stage of the disease currently lack an effective treatment to improve vision or slow disease progression.

According to a news release, an investigation is set to launch on SpaceX’s 30th Commercial Resupply Services (CRS) mission to the International Space Station (ISS) contracted by NASA to test the therapy in space. This could open new avenues for treating vision loss with this novel ocular gene therapy. Experiments on Earth have shown Oculogenex’s therapy is effective against retinal damage in models of retinal degeneration, indicating the potential to prevent progression to advanced AMD. However, these models do not emulate intermediate AMD’s milder, chronic oxidative stress.1

To address this, an ISS National Laboratory-sponsored investigation by Oculogenex will validate spaceflight as a novel biologic model of intermediate AMD. It will allow the company to evaluate its therapy in the space environment, providing an unparalleled opportunity to replicate a hard-to-study stage of the condition. According to the news release, the study will determine whether gene therapy can thwart spaceflight-induced retinal dysfunction and degeneration.

“The microgravity investigation could pave the way for future clinical trials and treatments, offering hope to those grappling with this debilitating condition,” Hema Ramkumar, CEO of Oculogenex and a physician treating people affected by macular degeneration in California, said in the news release.

According to researchers, the gene therapy boosts cellular response to oxidative stress and prevents retinal cell death, an approach that aims to restore the functionality of damaged cells and prevent vision loss.

“As we age, an epigenetic switch stops cellular repair, leading to cell degradation,” Ramkumar explained. “Our gene therapy renews dormant retinal stem cells, enhances mitochondrial function, and prevents cell senescence and death,” she said. “By targeting this epigenetic switch, our therapy instructs cells to remain active and resilient, promoting repair and sustaining vision.”

Separating itself from traditional therapies that typically address singular factors, Oculogenex’s gene therapy will target the root causes of dry macular degeneration at the intermediate stage, for which there are currently no treatment options.

According to the ISS National Lab news release, female rodents on the space station will be used to conduct this experiment, considering that macular degeneration disproportionately affects women. Spaceflight will serve as a disease model for age-related macular degeneration, with oxidative stress induced by spaceflight 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,” Ramkumar pointed out. “We anticipate our transformative gene therapy strategy will translate into improved and preserved vision for the millions living with dry macular degeneration.”

The company noted in the news release it received funding for this investigation through the Technology in Space Prize, funded by Boeing and the Center for the Advancement of Science in Space, manager of the ISS National Lab, in partnership with the MassChallenge startup accelerator program. Since its inception, the prize has provided approximately $20 million in funding to 32 projects for R&D sponsored by the ISS National Lab.

NASA’s SpaceX CRS-30 mission is scheduled to launch from Cape Canaveral Space Force Station in mid-March. This mission will include more than 40 ISS National Lab-sponsored payloads.

1. ISS National Lab-sponsored study tests a novel gene therapy for vision loss. EurekAlert! Accessed March 15, 2024.
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