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DAEJEON, South Korea – In a potentially landmark development for vision restoration, researchers at the Korea Advanced Institute of Science and Technology (KAIST) have developed a novel treatment capable of regenerating retinal nerves and restoring vision in animal models. Announced today, the findings offer significant hope to the over 300 million people globally at risk of vision loss from currently incurable retinal diseases.
Unlike existing treatments that primarily aim to slow disease progression, the new therapy, spearheaded by Professor Jin Woo Kim from KAIST’s Department of Biological Sciences, targets the underlying inability of mammalian retinas to repair themselves.
The research, published on March 25th in Nature Communications, identifies a key protein, PROX1 (prospero homeobox 1), as a major roadblock to natural retinal repair in mammals. While cold-blooded animals like fish can regenerate retinal tissue after injury via Müller glia cells, mammalian Müller glia are inhibited. Professor Kim’s team discovered that in damaged mammalian retinas, PROX1 accumulates in Müller glia, suppressing their regenerative potential. Crucially, they found this PROX1 isn’t produced by the glia themselves but is absorbed from surrounding damaged neurons that fail to degrade the protein.
Leveraging this discovery, the researchers developed an antibody designed to intercept and neutralize extracellular PROX1 before it can be taken up by the Müller glia. This antibody was developed in collaboration with Celliaz Inc., a biotech startup founded by Professor Kim’s lab.
In experiments using mouse models of retinal disease, including retinitis pigmentosa, administration of the PROX1-neutralizing antibody successfully triggered significant neural regeneration within the retina. Remarkably, delivering the antibody gene directly to the retina led to sustained regeneration and vision restoration lasting for over six months, marking the first instance of long-term retinal repair achieved in a mammal.
“Studies on fish have shown that retinal injuries trigger Müller glia to become progenitor cells, generating new neurons,” explained Professor Kim. “In mammals, this process is blocked. Our work identified PROX1 as the key inhibitor and demonstrated that blocking its transfer between cells can unlock the retina’s regenerative potential.”
Celliaz Inc. is now actively developing this antibody therapy, designated CLZ001, for human use against a range of degenerative retinal diseases lacking effective treatments.
“We are completing the optimization of the PROX1-neutralizing antibody (CLZ001) and preparing for preclinical studies,” stated Dr. Eun Jung Lee, involved in the research and development. “Our ultimate goal is to provide a tangible solution for patients facing blindness who currently have limited or no therapeutic options.”
The company is aiming to initiate human clinical trials by 2028, potentially paving the way for the first treatments capable of not just halting, but reversing vision loss from retinal damage.
Disclaimer: The research described in this article is based on preclinical studies conducted in animal models (mice). While promising, the findings have not yet been validated in humans. The therapy (CLZ001) is currently under development and requires further preclinical testing and successful human clinical trials to demonstrate safety and efficacy before becoming available as a treatment.
