GC Green Cross Publishes Preclinical Rare Disease Results Using Proprietary LNP Platform in International Journal

Demonstrating Superior Tolerability Compared to Conventional LNPs

GC Green Cross announced on September 4 that the results of its preclinical research on rare diseases, based on its independently developed LNP platform, have been published in the SCIE-level international journal 'Molecular Therapy-Nucleic Acids.'

Exterior view of GC Green Cross headquarters in Yongin, Gyeonggi Province. GC Green Cross

Currently, most mRNA-LNP therapeutics and vaccines that are either on the market or in clinical stages face limitations in being developed for chronic diseases due to issues such as liver toxicity and excessive immune responses upon repeated dosing. To overcome these challenges, GC Green Cross has established a lipid library using artificial intelligence (AI) and structure-activity relationship analysis, enabling the company to develop its own proprietary LNP platform for selecting optimal LNPs.

The company explained that both the mRNA and LNP used in this research were developed in-house, and their efficacy and safety were verified by applying them to animal models of phenylketonuria (PKU) and succinic semialdehyde dehydrogenase deficiency (SSADHD).

In animal studies, the LNPs from GC Green Cross were delivered to the liver and demonstrated superior protein expression compared to LNPs already validated in clinical trials. They also effectively reduced toxic metabolic substances that cause these diseases. Furthermore, even after repeated administration, there was no observed increase in liver enzyme (ALT, AST) levels or excessive immune responses, demonstrating excellent tolerability.

Additionally, by designing the optimal LNP composition, the company achieved not only batch-to-batch manufacturing reproducibility but also stability during frozen storage and freeze-thaw cycles, demonstrating its competitiveness in formulation quality.

Building on these results, GC Green Cross plans to expand its mRNA-LNP research pipeline to include chronic inflammatory diseases, cancer immunotherapies, and vaccines, as well as rare genetic disorders. The company is also exploring joint development and technology transfer opportunities with global pharmaceutical companies, leveraging its capabilities to independently produce both mRNA and LNP and its proprietary mRNA-LNP platform.

Jaeuk Jung, head of R&D at GC Green Cross, stated, "This research is significant in that it not only demonstrates the establishment of an LNP platform with excellent efficacy and safety through the structural design of ionizable lipids, but also increases the potential for developing therapeutics for rare diseases. We will further enhance our global competitiveness by advancing our next-generation LNP platform and expanding the application of mRNA therapeutics."