Revolutionary scientific research conducted in the unique conditions of low Earth orbit offers new hope to millions of people worldwide facing vision loss. In a surprising fusion of space technology and advanced medicine, scientists have utilized the environment of the International Space Station (ISS) to test and validate the potential of an innovative gene therapy for treating one of the leading causes of blindness – age-related macular degeneration (AMD).

This medical breakthrough not only promises to halt the progression of the disease, but initial results have also shown the possibility of restoring damaged retinal cells, which could mean the return of vision for patients who have already begun to lose it. At the heart of this success is the biotechnology company Oculogenex and its pioneering gene therapy targeting the BMI1 gene, which has shown outstanding results in conditions that perfectly simulate the degenerative processes in the eye.

What is macular degeneration and why is it a global problem?

Age-related macular degeneration is the leading cause of severe and irreversible vision loss in people over the age of 55 in the developed world. The disease affects the macula, the central and most sensitive part of the eye's retina. The macula is crucial for sharp, central vision that allows us to read, recognize faces, drive a car, and notice fine details. When the macula deteriorates, central vision becomes blurry, distorted, or dark spots appear in it, while peripheral vision usually remains preserved. This means a person can see the outline of a clock, but not the hands showing the time.

There are two basic forms of this disease:

• Dry (atrophic) macular degeneration: This is the most common form, accounting for about 80-90% of all cases. It is characterized by the slow, gradual breakdown of light-sensitive cells in the macula and the accumulation of protein deposits called drusen. It can progress for years, and for its early and intermediate stages, there is currently no effective treatment to stop its progression. Vision loss is gradual but can lead to significant impairment.


• Wet (exudative) macular degeneration: Although rarer, this form is responsible for about 90% of cases of severe vision loss associated with AMD. In the wet form, abnormal blood vessels begin to grow under the retina; they are fragile and prone to leaking fluid and blood. This leakage causes rapid and severe damage to the macula, leading to a sudden and dramatic loss of central vision. Existing treatments, such as anti-VEGF injections, can slow progression but require regular and unpleasant applications directly into the eye and do not offer a permanent solution.

Given the aging population, the number of affected individuals is predicted to continue to grow, making the search for a more effective and long-lasting treatment one of the priorities of modern ophthalmology.

The space station as a perfect laboratory for mimicking the disease

At first glance, the connection between space exploration and treating an eye disease on Earth may seem unusual. However, the environment on the International Space Station provides unique conditions that cannot be replicated in terrestrial laboratories. During long-duration missions, astronauts experience physiological changes that are surprisingly similar to the aging processes on Earth, only they occur much faster.

One of these changes is a phenomenon known as Spaceflight Associated Neuro-ocular Syndrome (SANS). In microgravity, body fluids redistribute and accumulate in the upper part of the body, including the head, which leads to increased intracranial pressure. This pressure strains the optic nerve and the back of the eye. In addition, exposure to space radiation creates an elevated level of oxidative stress. The combination of these two factors – increased pressure and oxidative stress – directly mimics the chronic stressors that cause retinal cell damage in patients with intermediate dry macular degeneration.

It was this realization that led scientists from Oculogenex to the idea of using the ISS as an ideal model for testing their gene therapy. Instead of waiting for years for the disease to develop in terrestrial models, in space they could study the effects of the therapy in an accelerated and highly disease-relevant environment.

A breakthrough gene therapy that regenerates cells

At the core of this research is an innovative gene therapy that targets a specific gene called BMI1. This gene belongs to the so-called "Polycomb" group of proteins and plays a crucial role in fundamental cellular processes, including DNA repair, mitochondrial function (the cell's power plants), and the self-maintenance and activation of stem cells. With age, the activity of the BMI1 gene weakens, leading to cellular aging (senescence) and a reduced ability of cells to resist damage.

The therapy from Oculogenex, developed under the leadership of ophthalmologist and retinal surgeon Dr. Hema Ramkumar, is designed as a one-time injection administered directly into the eye. The therapy introduces a functional copy of the BMI1 gene into the retinal cells, thereby triggering a process that can be described as "epigenetic reprogramming." In practice, this means that the therapy stimulates the cells in several key ways:

• Increases resistance to stress: Cells become more capable of resisting oxidative damage.


• Promotes repair: It activates mechanisms for repairing damaged DNA.


• Activates dormant stem cells: It potentially awakens dormant stem cells within the retina to replace lost or damaged tissue.


• Prevents cell death: It inhibits the process of programmed cell death (apoptosis) and aging (senescence), extending the lifespan of key retinal cells.

The goal of the experiment on the ISS was to assess how effectively this therapy can protect the retina in the extreme conditions of spaceflight. Using genetically treated mice, the team monitored their retinal function during exposure to microgravity and radiation.

Results that exceeded all expectations

The outcome of the research on the International Space Station was extraordinary. The data showed that the BMI1 gene therapy not only successfully prevented further damage to the retinal cells in mice exposed to space conditions but also achieved something the researchers had not hoped for in such a short time: significant regeneration and restoration of cells that were already showing signs of degeneration were observed.

This finding represents a huge step forward. While most existing therapies focus exclusively on slowing the disease, the results from space suggest that this gene therapy could also have regenerative potential, meaning the ability to repair existing damage. The data collected during the mission provided insights that would have required significantly longer and more expensive studies on Earth.

The success confirmed not only the effectiveness of Oculogenex's therapy but also the incredible value of the International Space Station as a platform for accelerating biomedical innovations. The results have generated great interest in the ophthalmological and biotechnological communities and have paved the way for the start of clinical trials in humans.

The future of treatment and new hope for patients

Successful testing in space marks a key milestone on the path to a potential cure for macular degeneration. For the millions of patients who currently face this diagnosis, especially those with intermediate dry macular degeneration for which there is no cure, this news brings concrete hope. The possibility of receiving a single injection that could provide long-term protection and even restore vision would change the treatment paradigm for this devastating disease.

Such a therapy would preserve the independence, mobility, and quality of life for patients, allowing them to continue with the activities they love. The success of this project, sponsored by the ISS National Lab, also highlights how investment in space research brings tangible benefits for solving medical challenges on Earth, opening new chapters in the fight against age-related diseases.