ISS National Lab-Sponsored Study Tests a Novel Gene Therapy for Vision Loss

CAPE CANAVERAL (FL), March 14, 2024 – Age-related macular degeneration (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. Now, a new gene therapy from biotech innovator Oculogenex, Inc. may change that narrative by preventing and even reversing symptoms of this pervasive condition.

Set to launch on SpaceX’s 30th Commercial Resupply Services (CRS) mission to the International Space Station (ISSInternational Space Station) contracted by NASANational Aeronautics and Space Administration, an investigation to test the therapy in space may 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.

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. The investigation seeks to ascertain whether gene therapy can thwart spaceflight-induced retinal dysfunction and degeneration.

“The microgravityThe condition of perceived weightlessness created when an object is in free fall, for example when an object is in orbital motion. Microgravity alters many observable phenomena within the physical and life sciences, allowing scientists to study things in ways not possible on Earth. The International Space Station provides access to a persistent microgravity environment. investigation could pave the way for future clinical trials and treatments, offering hope to those grappling with this debilitating condition,” said Hema Ramkumar, CEO of Oculogenex and a physician treating people affected by macular degeneration in California.

The gene therapy enhances 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,” said Ramkumar. “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.”

Distinguishing itself from conventional 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.

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.

Ramkumar explained, “By harnessing the distinctive conditions of spaceflight, we aim to gain invaluable insights into the potential of spaceflight as a model of dry AMD. We anticipate our transformative gene therapy strategy will translate into improved and preserved vision for the millions living with dry macular degeneration.”

Oculogenex received funding for this investigation through the Technology in Space PrizeA prize that provides grant funding for business startups participating in the MassChallenge startup accelerator program to conduct innovative research and technology development utilizing the ISS National Lab. The prize is funded by the Center for the Advancement of Science in Space, which manages the ISS National Lab, and Boeing., 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 slated to launch from Cape Canaveral Space Force Station in mid-March. This mission will include more than 40 ISS National Lab-sponsored payloads. To learn more about all ISS National Lab-sponsored research on this mission, please visit our launch page.

Download a high-resolution photo for the release: Oculogenex Research Image

Media Contact:       
Patrick O’Neill
904-806-0035
PONeill@ISSNationalLab.org

# # #

About the International Space Station (ISS) National Laboratory: The International Space Station (ISS) is a one-of-a-kind laboratory that enables research and technology development not possible on Earth. As a public service enterprise, the ISS National Laboratory® allows researchers to leverage this multiuser facility to improve quality of life on Earth, mature space-based business models, advance science literacy in the future workforce, and expand a sustainable and scalable market in low Earth orbit(Abbreviation: LEO) The orbit around the Earth that extends up to an altitude of 2,000 km (1,200 miles) from Earth’s surface. The International Space Station’s orbit is in LEO, at an altitude of approximately 250 miles.. Through this orbiting national laboratory, research resources on the ISS are available to support non-NASA science, technology, and education initiatives from U.S. government agencies, academic institutions, and the private sector. The Center for the Advancement of Science in Space™ (CASIS™(Abbreviation: CASIS™) The nonprofit organization that manages the ISS National Lab, which receives at least 50 percent of the U.S. research allocation on the International Space Station to facilitate research that benefits humanity (NASA manages the other 50% and focuses on research for space exploration purposes).) manages the ISS National Lab, under Cooperative AgreementA cooperative agreement is Federal assistance that establishes a relationship between the U.S. Government and a recipient in which the principal purpose of the relationship is to accomplish a public purpose of support or stimulation.  Since 2011, the Center for the Advancement of Science in Space™ (CASIS™) has managed the National Laboratory® through a Cooperative Agreement with NASA. with NASA, facilitating access to its permanent microgravity research environment, a powerful vantage point in low Earth orbit, and the extreme and varied conditions of space. To learn more about the ISS National Lab, visit our website.

As a 501(c)(3) nonprofit organization, CASIS accepts corporate and individual donations to help advance science in space for the benefit of humanity. For more information, visit our donations page.