KAIST researchers have announced the development of a groundbreaking drug aimed at restoring vision lost due to retinal diseases, with details revealed on March 30, 2025.

This innovative antibody, developed by Celliaz Inc., has shown remarkable efficacy in promoting neural regeneration in treated mice, leading to sustained vision restoration in models of retinitis pigmentosa for over six months.

The treatment resulted in significant vision recovery, marking a pivotal breakthrough in the field of mammalian retinal regeneration.

The research team, led by Professor Jin Woo Kim, developed a method to enhance the regenerative ability of Müller glia by utilizing an antibody to neutralize extracellular PROX1, a known inhibitor of retinal regeneration.

Their study identified the PROX1 protein as a major barrier to Müller glia dedifferentiation in mammals, revealing that it accumulates in damaged retinal cells.

Interestingly, the research demonstrated that PROX1 is not produced by Müller glia; instead, it is sourced from surrounding neurons that secrete the protein.

Dr. Eun Jung Lee highlighted the goal of optimizing the PROX1-neutralizing antibody (CLZ001) for preclinical studies, aiming to assist patients at risk of blindness.

This research is particularly significant as over 300 million people worldwide are at risk of vision loss due to retinal diseases, with currently no available treatments to restore damaged retinas.

Unlike cold-blooded animals such as fish, which can regenerate retinal neurons through Müller glia cells, mammalian retinas have a limited capacity for regeneration after damage.

Celliaz Inc. is committed to further developing this retinal regeneration therapy and plans to initiate clinical trials by 2028.