Skip to Content

Mirna Therapeutics Publishes Preclinical Data Demonstrating Therapeutic Effects of microRNA in Hematological Cancer

AUSTIN, Texas--(BUSINESS WIRE)--May 2, 2012 - Mirna Therapeutics, Inc. announced today the publication of new results in the journal Leukemia demonstrating that the therapeutic delivery of microRNA-34 (miR-34) mimics inhibits tumor growth in an animal model of lymphoma.

The results were generated in collaboration with Prof. Dr. Anne Müller at the University of Zurich, Switzerland, who investigates the role of miRNAs in lymphoma. Previously, she showed that Myc-mediated repression of miR-34 promotes the high-grade transformation of B-cell lymphoma and suggested that miR-34 replacement is a promising strategy to combat this aggressive and very difficult to treat type of cancer. The current work now describes that the systemic administration of formulated miR-34 mimics effectively reduces tumor growth in an animal model of B-cell lymphoma.

“The data indicate that the therapeutic utility of miR-34 mimics is not limited to solid tumor types and can also be applied to hematological cancers,” says Dr. Paul Lammers, President & CEO. “This speaks to the important role of miR-34 during tumor suppression and its widespread inactivation in many other cancer types.” The data complement those generated by Mirna scientists and academic collaborators in animal models of lung and prostate cancer (Wiggins et al., Cancer Research, 2010; Trang et al., Molecular Therapy, 2011; Liu et al., Nature Medicine, 2011).

miR-34 is a non-coding RNA that functions as a powerful tumor suppressor and is lost or downregulated in a majority of human cancers. Mirna Therapeutics has initiated a pre-clinical development program to advance a miR-34 therapy to the clinic, and aims to initiate its first Phase 1 clinical trial early 2013. This work is one of the first to describe a therapeutic miRNA approach for leukemias and lymphomas.

This project was funded in part by a Cancer Prevention and Research Institute of Texas (CPRIT) Commercialization grant.

About microRNA

microRNAs (miRNAs) are approximately 20-25 nucleotides long and affect gene expression by interacting with messenger RNAs. Unlike siRNAs, miRNAs are encoded in the human genome and are used as natural regulators of global gene expression. More than 1,500 miRNAs are encoded in the human genome and comprise approximately 2% of all mammalian genes. Since each miRNA appears to regulate the expression of tens to hundreds of different genes, miRNAs can function as “master-switches,” efficiently regulating and coordinating multiple cellular pathways and processes. By coordinating the expression of multiple genes, miRNAs are responsible for guiding proper embryonic development, immunity, inflammation, as well as cellular growth and proliferation. Misregulation of miRNAs appears to play a fundamental role in the occurrence, growth and dissemination of many cancers, and replacement of down regulated miRNAs in tumor cells results in a positive therapeutic response.

About Mirna Therapeutics

Mirna Therapeutics, Inc. (Mirna) is a biotechnology company focused on the development and commercialization of miRNA therapeutics. The Company has a foundational intellectual property portfolio on the therapeutic use of miRNAs developed by its own scientists as well as in-licensed from other institutions. Mirna's IP portfolio contains >300 miRNAs with applications in oncology and other diseases. Oncology-directed miRNAs include those that are key tumor suppressors in cancer, such as miR-34 and let-7 that have been shown to block tumor growth in a number of different pre-clinical animal studies. The Company, founded in 2007 and located in Austin, Texas, has received significant funding from the State of Texas, both through the State's Emerging Technology Fund and from the Cancer Prevention and Research Institute of Texas (CPRIT). Mirna Therapeutics is the recipient of a $10.3 million commercialization award from CPRIT. For more information, visit


Contact: Mirna Therapeutics, Inc.
Paul Lammers, M.D., M.Sc., 512-901-0909
President and CEO



Posted: May 2012