Addressing the Limitations of Existing Animal Models and the Need for Human-Centric Biological Data

Historically, drug development has heavily relied on animal models, such as mice. However, due to biological differences between species, clinical trial failure rates have been high. Complex diseases like liver fibrosis and metabolic dysfunction-associated steatohepatitis (MASH) pose a significant challenge, as animal studies often fail to accurately predict clinical improvements in humans. To overcome these limitations, there is a growing emphasis on human-first datasets, which prioritize the use of patient-derived genomic and transcriptomic data. This shift represents a paradigm change in the industry, aiming to maximize clinical prediction and reduce development costs by incorporating real human data from the initial hypothesis generation stage.

Ochre Bio and Lexogen's Large-Scale Liver Gene Analysis Collaboration

Ochre Bio, a UK-based biotechnology company, and Lexogen, an Austrian transcriptomics specialist, have successfully established a large-scale functional genomics dataset for liver diseases, utilizing over 120,000 primary human hepatocytes. In this collaboration, Lexogen provided its high-resolution gene expression profiling technology, which does not require an extraction process, enabling Ochre Bio to acquire high-quality RNA-Seq data. The collaboration goes beyond simply outsourcing sequencing services; it provides precise data that can be immediately recognized and interpreted by artificial intelligence (AI) machine learning, significantly improving the computational efficiency of OBELiX, Ochre Bio's target discovery platform.

Uncovering Causal Relationships and Innovating Target Discovery Through Machine Learning

Unlike traditional genomic data, which primarily focuses on identifying statistical correlations, this dataset emphasizes uncovering direct causal relationships by combining gene perturbation experiments, such as gene knockout. Ochre Bio utilizes liver tissue donated from Asian regions and a New York-based ex vivo liver perfusion system to maintain the entire human liver in a living state, allowing for direct measurement of physiological changes. By combining this experimental data with AI in a 'lab-in-the-loop' approach, the company can highly accurately predict therapeutic targets that can induce the expression of fibroblast growth factor 21 (FGF21), a key factor in mitigating liver fibrosis, from a vast number of genes.

Commercialization Prospects in the Global MASH Market and Benefits from Regulatory Changes

Following the approval of Madrigal Pharmaceuticals' Rezdiffra (resmetirom) as the first MASH treatment by the U.S. Food and Drug Administration (FDA) in March 2024, the global market for related therapeutics is expected to grow rapidly to $57 billion (approximately 78 trillion KRW) by 2035. In line with this, the FDA announced in April 2025 guidelines that encourage the use of alternative testing methods, such as computer modeling and organoids, and reduce the mandatory use of animal experiments in the drug development process, leading regulatory changes. Ochre Bio, in the pre-clinical stage, has secured partnerships with Boehringer Ingelheim for up to $1 billion and a co-licensing agreement with GSK, demonstrating significant value from its exclusive data assets from an early stage.