Our vision, arguably one of our most vital senses, is under threat for over 300 million people globally due to a range of retinal diseases. Despite significant advancements in treatments for these diseases, the ability to restore vision that has already been lost has remained elusive – until now. Researchers at KAIST have made a significant breakthrough in the development of a drug that could potentially restore vision.
The announcement was made on March 30th by KAIST, represented by President Kwang Hyung Lee, detailing how Professor Jin Woo Kim’s team from the Department of Biological Sciences achieved this remarkable feat. They created a method to reinvigorate vision by regenerating retinal nerves.
The team managed to trigger retinal regeneration and the recovery of vision in a disease-model mouse. This was achieved by blocking the PROX1 protein, known for inhibiting retinal regeneration, using a specific compound. This effect was sustained for over six months, marking an unprecedented victory in long-term neural regeneration in mammalian retinas.
Retinal diseases are becoming more prevalent as the world’s population ages. However, restoring damaged retinas and vision remains a challenge due to the mammalian retina’s inability to regenerate after damage. This differs from cold-blooded animals, like fish, which can regenerate their retinas following injury.
The research team identified the PROX1 protein as a critical factor hindering the ability of Müller glia cells to revert to retinal progenitor cells in mammals. In contrast, fish display an abundance of regenerative Müller glia which lack the PROX1 protein, suggesting it inhibits retinal regeneration.
It was found that the PROX1 protein doesn’t originate from within the Müller glia cells of the damaged mouse retina. Instead, it is taken from neighbouring neurons which fail to break down the protein and secrete it instead.
The team capitalised on this discovery and developed a solution to restore the regenerative ability of Müller glia cells. They used an antibody that binds to PROX1, developed by biotech startup Celliaz Inc., founded by Professor Jin Woo Kim’s research lab. This antibody, when applied to the retinas of disease-model mice, significantly boosted neural regeneration and, when delivered to the retinas of mice with retinitis pigmentosa, it enabled continuous retinal regeneration and vision restoration for over half a year.
Celliaz Inc. is now working to develop this retinal regeneration-inducing therapy for application in various degenerative retinal diseases that currently lack effective treatments. The company’s goal is to begin clinical trials by 2028.
Dr. Eun Jung Lee stated, “We are about completing the optimization of the PROX1-neutralizing antibody (CLZ001) and move to preclinical studies before administering it to retinal disease patients. Our goal is to provide a solution for patients at risk of blindness who currently lack proper treatment options.”
The study received support from the Korean National Research Foundation (NRF) and the Korea Drug Development Foundation (KDDF).
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