Development of a global innovative drug in eye drop form for treating dry age-related macular degeneration

Age-related macular degeneration (AMD) is the leading cause of vision loss in individuals over 65, characterized by abnormal changes in the macular, resulting in reduced vision and distorted objects. Dry AMD accounts for 90% of all AMD cases, with relatively mild vision impairment; however, approximately 30% progress to the severe vision loss associated with wet AMD within 10 years. The only FDA-approved treatments for dry AMD as of 2023 are two injectable drugs, which are limited by concerns over complications from intravitreal injections and modest efficacy in restoring vision.

The research team led by Dr. Moon-Hyeong Seo from the Natural Product Drug Development Center at the Korea Institute of Science and Technology (KIST, President Oh Sang-Rok) has developed a new therapeutic agent for dry AMD that can be administered as eye drops. Eye drops are the most preferred drug delivery method in the ophthalmic market, yet developing eye drop formulations targeting the retina, located in the posterior segment of the eye, remains a significant challenge.

To address the limitations of injection-based treatments, the research team focused on the inflammatory signaling pathway of Toll-like receptors (TLRs), which are known to play a critical role in AMD pathogenesis. By extracting peptide sequences from tens of thousands of proteins with structures similar to natural TLR signaling proteins, they established an extensive library of over 190,000 peptide drug candidates. Utilizing advanced technology for rapidly screening peptides that specifically bind to TLR signaling proteins, they successfully identified multiple candidate peptides capable of inhibiting interactions between these proteins.

The researchers validated the therapeutic efficacy of the peptides by administering them as eye drops to mice with induced dry AMD. The treated group exhibited retinal cell protection and significantly reduced retinal degeneration, comparable to normal mice. This demonstrated that peptide-based eye drops could effectively replace existing injectable therapies for dry AMD.

This new therapeutic agent, delivered in eye drop form, offers enhanced treatment convenience and adherence for patients while reducing complications and costs associated with repetitive invasive treatments. Furthermore, the non-invasive and safe nature of the therapy provides a novel treatment option that improves both efficacy and patient satisfaction. This innovation is expected to revolutionize the treatment accessibility of AMD and other related ophthalmic conditions.

Dr. Seo stated, “The KIST Natural Product Drug Development Center, established in September to focus on mission-driven research, aims to develop global drugs targeting aging-related diseases, including cancer and ophthalmic conditions. We plan to pursue collaborative research with domestic and international pharmaceutical companies to advance global clinical trials for this innovative dry AMD therapeutic.”

###

KIST was established in 1966 as the first government-funded research institute in Korea. KIST now strives to solve national and social challenges and secure growth engines through leading and innovative research. For more information, please visit KIST’s website at https://eng.kist.re.kr/

This research was supported by the Ministry of Science and ICT (Minister Sang Im Yoo) through KIST's major project and the Excellent Young Researcher Program (2021R1C1C1003843). The findings were published as an inside back cover article in the international journal 「Advanced Science」 (IF 14.3, JCR category top 6.5%).