KAIST Develops World’s First Vision-Restoring Drug
- Yul So
- Apr 3
- 2 min read
April 3, 2025
By Yul So
A domestic research team has successfully developed a new drug that can restore damaged vision for the first time in the world. KAIST (Korea Advanced Institute of Science and Technology) announced on March 30, that a research team led by Professor Jinwoo Kim from the Department of Biological Sciences has developed a treatment method that can restore vision through retinal nerve regeneration. The team successfully included retinal tissue regeneration by blocking the PROX1 protein, which inj=hibits retinal repair, and confirmed that the treatment’s effects lasted for more than six months in disease-model mice.
This breakthrough marks the world’s first successful case of long-term nerve regeneration in mammalian retains, offering new hope for patients suffering from degenerative retinal diseases, for which no effective treatments currently exist. With the global aging population, the number of retinal disease patients continues to rise, yet vision restoration remains a challenge due to the reina’s limited regenerative ability.
The team identified PROX1 as a key factor preventing Müller glia, a type of retinal cell, from regenerating damaged neurons. In contrast to fish, where retinal regeneration occurs naturally, PROX1 accumulates in the Müller glia of mammals, blocking their ability to dedifferentiate into neural progenitor cells. The study revealed that PROX1 in mammalian Müller glia is not produced internally but is instead secreted by surrounding neurons that fail to degrade it.
To overcome this barrier, the researchers developed a method to remove PROX1 externally before it reaches the cells, restoring Müller glia’s regenerative ability. The treatment, utilizing a high-affinity antibody targeting PROX1, successfully induced nerve regeneration in disease-model mice. When delivered as a gene therapy to mice with congenital retinal degenerative disease, it sustained continuous neuron generation and vision recovery for over six months. KAIST startup Celiaz Inc. is currently developing the therapy for various degenerative retinal diseases, aiming to begin clinical trials by 2028.
Dr. Eunjeong Lee from the research team stated, “We are finalizing efforts to enhance the efficacy of the PROX1-neutralizing antibody (CLZ001), and after completing efficacy and safety evaluations in various animal models, we aim to administer it to retinal disease patients.”
The study was published in Nature Communications on March 26.