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Small Molecule Inhibitors of MicroRNA miR21

Tech ID

Micro RNAs are non-coding RNAs that appear to regulate gene expression. Numerous microRNAs are implicated in various diseases, including cancer, and provide new targets for therapeutic intervention.


Researchers at The Wistar Institute have developed a high-throughput screening (HTS) assay to identify inhibitors of oncogenic microRNAs. Using chemical libraries and the HTS assay, Wistar scientists (in collaboration with Dr. Alex Dieters at North Carolina State University) were able to identify several compounds capable of reducing the expression of the microRNA miR-21. This assay system may be used to find activators or inhibitors of other microRNAs.


Using the results of the HTS assay, the researchers designed and synthesized group of small molecules that selectively inhibit the microRNA miR21. MiR-21 functions as an anti-apoptopic agent in cancer cells and elevated levels of miR-21 has been associated with breast, ovarian, lung, and brain cancers and with heart failure. These compounds may be particularly useful in combination with low doses of standard chemotherapeutic drugs. Additional studies to demonstrate the in vivo efficacy of these compounds are planned.

Key Words
Cancer, cancer treatment, hear failure, micro-RNA, miR-21, oncology, small molecule therapeutic
Applications and Advantages

This technology is useful for developing first-in-class selective microRNA inhibitors for treatment of cancer and heart failure. The small molecule miR-21 inhibitors that have been developed using the technology may be candidates for treatment of these diseases, either alone or in combination with known drugs.

Intellectual Property Status

PCT Application No. PCT/US2009/034611, filed 02/20/2009.

Licensing Opportunity

Wistar is seeking corporate partners to collaborate in (i) the validation and development of the miR21 inhibitors compounds, and (ii) the identification of new microRNA inhibitors using our novel assay system. An exclusive license or sponsored research agreement would be considered.

Relevant Publication(s)

Gumireddy, K., Young, D. D., Xiong, D., Hogenesch, J. B., Huang, Q., Deiters, A. (2008) “Small Molecule Inhibitors of MicroRNA miR-21 Function”. Angewandte Chemie International Edition 47(39):7482-7484.