[Hinews] SEOUL, South Korea — Seoul National University Hospital (SNUH) has been selected as the lead institution for South Korea’s 2025 ARPA-H-inspired initiative, aimed at developing a tailored treatment platform for pediatric rare diseases and conducting pioneering N-of-1 clinical trials. The project, backed by the Ministry of Health and Welfare, the Korea Health Industry Development Institute, and the K-Health Future Promotion Agency, will receive up to 14.75 billion KRW (approximately $11 million USD) over 4.5 years.

SNUH will collaborate with Sookmyung Women’s University, the National Institute of Toxicological Research, Kyung Hee University, and STpharm Co., Ltd., forming a consortium to revolutionize the treatment paradigm for pediatric rare diseases. The initiative seeks to address the urgent need for therapies for the roughly 7,000 rare diseases, 80% of which manifest in childhood.

Exterior view of Seoul National University Hospital (Image source: Seoul National University Hospital website)
Exterior view of Seoul National University Hospital (Image source: Seoul National University Hospital website)
While advancements in genomic medicine and institutional support have improved diagnostic rates, treatments remain scarce. Severe pediatric rare diseases, in particular, face significant hurdles, as traditional drug development often takes over a decade, limiting therapeutic options. Globally, there is growing emphasis on rapid, patient-specific drug development tailored to individual genetic mutations.

The SNUH-led consortium will leverage antisense oligonucleotide (ASO) technology to design and produce personalized therapies. ASOs bind to specific mRNA sequences to modulate protein expression, targeting the root causes of diseases. This approach is at the forefront of precision medicine research. The project also marks South Korea’s first foray into N-of-1 clinical trials, designed to develop and test therapies for individual patients.
This research is poised to establish a novel treatment platform for children with severe rare diseases, with potential for broader application to patients with diverse genetic mutations. The initiative aims to enhance treatment access for rare disease patients in South Korea and position the country as a leader in global precision medicine.

Dr. Jong-Hee Chae, the lead investigator and a professor of clinical genomics at SNUH, emphasized the project’s significance. “This will be a turning point, offering new hope to children suffering from rare diseases and their families,” Chae said. “We are committed to ensuring that personalized gene therapies can be applied to real-world patients.”

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