Poster
HUGO-GT™ Models：Efficacy Data & Core Advantages for Small Nucleic Acid Drug Therapeutics
HUGO-GT™(Humanized Genomic Ortholog for Gene Therapy) is a next-generation platform designed to replace full-length human genomic sequences-including promoters, exons, introns, and untranslated regions (UTRs)-at thecorresponding mouse locus. This approach establishes a highly human-relevant preclinical research environment and provides a robust platform for the development of genetic and nucleic acid therapies.
Overview
The HUGO-GT™ platform is an advanced humanized genomic model platform designed to improve the clinical relevance of preclinical research. By accurately replicating complex human gene regulatory networks, it enables more predictive evaluation of RNA-targeting therapeutics, including ASOs, siRNAs, and gene-editing approaches. Beyond rare diseases, HUGO-GT™ supports research across major therapeutic areas such as cardiovascular, metabolic, neurodegenerative, oncology, and autoimmune diseases. Powered by TurboKnockout™ technology, the platform delivers ultra-precise genomic engineering with the ability to replace genomic regions up to 500 kb, providing stable, reliable models for gene function studies, drug discovery, and efficacy evaluation.
Key Insights
This poster presents:
- Clinically Predictive Models: Faithfully recapitulates human gene regulation to improve the translational relevance of ASO, siRNA, and gene-editing research.
- Broad Therapeutic Applications: Supports drug discovery and target validation across rare diseases, oncology, cardiovascular, metabolic, neurodegenerative, and autoimmune disorders.
- Advanced Genome Engineering: TurboKnockout™ technology enables high-precision replacement of genomic regions up to 500 kb, delivering robust and reliable humanized models.
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