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Development of Novel Therapeutic Agent for LKB1-Inactivated Lung Cancer Targeting SMOC1 with Oligonucleotide

An innovative therapeutic approach for refractory LKB1-inactivated lung cancer using next-generation siRNA targeting specifically overexpressed SMOC1.

Advantages

- Addressing Unmet Medical Needs: The first specific treatment for "immune-cold" LKB1 (STK11) -inactivated lung cancer, which is resistant to existing EGFR inhibitors and Immune Checkpoint Inhibitors (ICIs).
- High Safety and Specificity: SMOC1 expression in adults is limited to the testes, minimizing the risk of systemic side effects.
- Highly Original Target: The link between SMOC1 and cancer is a unique discovery by the inventor, with no competing development projects currently identified.

Current Stage and Key Data

- Validated In Vitro: Confirmed cell proliferation and angiogenesis inhibition through SMOC1 knockdown in tumor cell lines with LKB1 loss-of-function mutations.
- Proven In Vivo Efficacy: Demonstrated significant tumor growth inhibition in mouse xenograft models.

Partnaring Model

- Preferred Partnership: Joint research and development, patent licensing.
- Potential Partners: Pharmaceutical companies with nucleic acid medicine platforms, biotech ventures seeking to expand anti-cancer pipelines, and companies specializing in DDS.

Background

Inactivating mutations of the LKB1gene are found in approximately 15-20% of non-small cell lung cancers. This subset of lung cancer exists exclusively from EGFR mutations and is characterized by its resistance to immune checkpoint inhibitors, a phenomenon known as being "immune-cold," making it a highly difficult-to-treat condition.
We discovered that SMOC1 is specifically overexpressed in LKB1-inactivated lung cancer, promoting tumor growth and angiogenesis. Targeting SMOC1 offers a promising new therapeutic strategy for patient populations that do not respond to current standard-of-care treatments.

Principal Investigator

Ichidai TANAKA (Department of Respiratory Medicine, Nagoya University Hospital, Tokai National Higher Educational and Research System)

Patents and Publications

Patent pending

Project No:bk-05220