3billion Joins Korean ARPA-H ‘ARISE’ Collaborative Research Project

“Expanding Rare Disease Genetic Diagnostics Expertise into ASO Therapeutics Research”

• 3billion selected as a joint R&D institute for a Korean ARPA-H project with up to KRW 8 billion in research funding
• Project aims to establish an AI- and patient data-enabled platform for ASO therapeutic development and complete preclinical validation
• 3billion to contribute AI-powered genomic interpretation technology and its rare disease patient network to ASO therapeutic development and validation

SEOUL, South Korea, September 22, 2026 — 3billion, an AI-powered rare disease genetic diagnostics company, announced that it has been selected as a joint research and development institute for the “Development and Validation of a Proactive N-of-Many ASO Therapeutics Platform for Rare Diseases” project under ARISE (Accelerating Rare Disease Innovation through Science and Evidence), a Korean ARPA-H initiative. The project is led by the Korea Advanced Institute of Science and Technology (KAIST), with 3billion and Asan Medical Center participating as joint R&D institutes.

The Korean ARPA-H Project is a national research and development initiative launched by the Ministry of Health and Welfare (MOHW) and implemented through the Korea Health Industry Development Institute (KHIDI) and its K-Health MIRAE Initiative to address major unmet challenges in healthcare. The research is being conducted as part of ARISE, an initiative under the Korean ARPA-H mission area of overcoming intractable diseases. ARISE aims to develop and validate precision therapeutics platforms for rare diseases by integrating AI with patient data. The project will run for four and a half years, from July 2026 through December 2030, with up to KRW 8 billion in R&D funding.

In the field of personalized ASO therapeutics, the N-of-1 approach, in which a treatment is developed specifically for a single patient, has been widely explored. However, because each patient requires an individualized development process, from preclinical studies through therapeutic development, the approach can be highly time- and resource-intensive. Its broader applicability and economic feasibility are also inherently limited, as the resulting therapy is intended for only one patient.

To overcome these limitations, the project will pursue an N-of-many strategy that proactively identifies groups of patients who share the same or similar genetic causes and develops therapies that may be applicable across those groups. By identifying patients with identical or similar genetic variants early in the platform development process, the project aims to substantially improve the efficiency of rare disease drug development and reduce the time required to move from diagnosis to treatment.

The project will use antisense oligonucleotides (ASOs) as a core therapeutic modality. ASOs are short, synthetic, single-stranded oligonucleotides designed to bind selectively to specific RNA targets associated with disease. ASOs can precisely regulate gene expression by suppressing the production of abnormal proteins or by modulating RNA splicing to promote the production of functional proteins.

Because of their high target specificity, ASOs are particularly well suited to the development of precision therapeutics. Building on this advantage, the project aims to establish a platform for the proactive identification and development of ASO therapeutic candidates and ultimately complete preclinical validation of selected candidates.

3billion will leverage its rare disease genomic and clinical data and AI-powered genetic variant interpretation technology to analyze patients’ genetic characteristics and clinical phenotypes, providing evidence to support the identification of appropriate treatment candidates. The company will also identify patients with the same or similar genetic variants across multiple medical institutions, helping to define the potential applicability and target population of each therapeutic approach. Through the project, 3billion plans to extend the use of its diagnostic data and medical network to rare disease therapeutic R&D and support the development of ASO therapeutics for broader patient populations.

Changwon Keum, CEO of 3billion, said, “In rare disease drug development, accurately interpreting disease-causing variants and identifying appropriate patient populations are critical. Building on these capabilities, we aim to bring the genomic and clinical data and AI interpretation expertise developed through rare disease diagnostics into therapeutic R&D. Through this collaboration, we hope to move beyond the traditional N-of-1 approach and create meaningful new treatment opportunities for more patients with rare diseases.”