Advantages

- Host-side drug target commonly exploited by multiple RNA virus infections
- GBF1 was identified from a screening of 91 host factors as a factor that strongly suppresses viral replication while maintaining high cell viability
- Reduces the risk of drug-resistant variants emerging due to viral mutation

Current Stage and Key Data

- Dozens of antisense oligonucleotides (ASOs) with low off-target risk and high binding affinity were designed, and ASOs with high GBF1 knockdown efficiency were selected.
- In vitro, GBF1-targeting ASOs strongly inhibited the replication of influenza virus and SARS-CoV-2 at low concentrations (IC50: 17.06–44.75 nM) and showed high selectivity (SI: 155–294.5).
- In vivo evaluation using RNA virus infection models is currently in progress.

Partnaring Model

We are seeking pharmaceutical companies and biotech ventures focusing on the development of nucleic acid therapeutics or antiviral agents for collaborative research and/or development toward practical application through patent licensing.

Background and Technology

RNA virus infections, including respiratory infections, viral hemorrhagic fevers, and zoonotic diseases, remain a major global health challenge. In contrast, many existing antivirals target viral proteins, and the emergence of drug-resistant variants driven by viral mutations has become a serious problem. Moreover, there are few broad-spectrum therapeutics that do not depend on a specific virus and can be rapidly deployed against unknown emerging infections, expecting the need for new therapeutic candidates.
This technology represents a novel antiviral strategy focusing on GBF1, a host factor commonly exploited for replication by many RNA viruses. By using newly designed antisense oligonucleotides to appropriately suppress host GBF1 expression, this approach is expected to provide a new therapeutic candidate that can inhibit the replication of diverse viruses with a single agent, irrespective of viral species or mutations.

Principal Investigator

Prof. Tokiko WATANABE (Research Institute for Microbial Diseases, The University of Osaka）

Patents and Publications

Patent application: Pending (unpublished)
Publication: Simanihuruk et al., iScience, 2026; 29
DOI: https://doi.org/10.1016/j.isci.2026.114851