Advantages
- Precise Functional Control: Distinct from earlier RNA-degrading (Gapmer), minimizes side effect risks by selectively inhibiting only the domains essential for cancer maintenance while preserving other physiological functions of the lncRNA.
- Breakthrough Mechanism: Exerts anti-tumor effects by preventing the resolution of harmful R-loop structures that accumulate in cancer cells, without requiring RNA degradation.
- Clinically Proven Design: Employs full-base chemical modifications used in established nucleic acid drugs (e.g., Nusinersen) to ensure high in vivo stability and tolerability.
Current Stage and Key Data
Animal POC Stage
- Target Site Identification: Identified the primary binding site for specific factors involved in R-loop resolution through in vitro assays.
- Proliferation Inhibition: Confirmed that modified oligonucleotides targeting this site significantly suppress cell growth without degrading the target RNA.
- In Vivo Efficacy: Demonstrated significant suppression of tumor growth and extended survival in mouse brain tumor models.
Partnaring Model
- Preferred Models: Exclusive/non-exclusive licensing or joint research and development.
- Potential Partners: Major pharmaceutical / Biotech companies focused on oligonucleotides, nucleic acid platform companies, and biotech firms with advanced DDS technologies.
Background and Technology
In intractable tumors such as pancreatic cancer and glioblastoma, cancer cells maintain their growth by skillfully resolving harmful R-loop structures generated during transcription to protect their DNA. Existing nucleic acid therapies primarily focus on the total degradation of lncRNAs involved in this process, which carries the risk of impairing physiological functions in normal tissues. By "sterically blocking" the binding site between the lncRNA and R-loop resolution factors, this technology specifically disrupts the cancer-specific survival strategy, aiming to achieve both high therapeutic efficacy and safety.
Principal Investigator
Yutaka KONDO (Division of Cancer Biology, Nagoya University Graduate School of Medicine, Tokai National Higher Education and Research System)
Patents and Publications
Patent pending