Tekmira Acquires Worldwide Exclusive License to Novel RNAi Platform

Tekmira Pharmaceuticals Corporation recently announced it has obtained an exclusive, worldwide license to a novel and proprietary RNAi technology called MV-RNA (multivalent RNA) from Halo-Bio RNAi Therapeutics, Inc. The exclusive license and collaboration agreement provides for the companies to work together to design and develop MV-RNA molecules to gene targets of interest to Tekmira and to combine MV-RNA molecules with Tekmira’s lipid nanoparticle (LNP) technology to develop therapeutic products.

“MV-RNA technology has tremendous potential to lead the future development of RNAi therapeutic products. The power of MV-RNA technology is its capacity to silence multiple molecular targets with a single molecule and treat disease by targeting multiple cellular pathways simultaneously, which clearly differentiates the technology from all other RNAi technologies currently available. Our exclusive license to Halo-Bio’s MV-RNA technology expands and diversifies our technology base to support the development of novel products and gives us the opportunity for broader discussions with the pharmaceutical industry,” said Dr. Mark J. Murray, Tekmira’s President and CEO.

Halo-Bio’s MV-RNA technology comprises single macromolecules capable of mediating RNAi at multiple unique target sites. MV-RNA can target three sites on a single gene or up to three separate genes simultaneously. Tekmira has successfully demonstrated multi-gene knockdown using MV-RNA enabled by proprietary LNP formulations.

“This transaction places Tekmira in the unique position of having the leading RNAi delivery technology and access to multiple RNA payload technologies to develop RNAi therapeutic drugs. We believe we can accelerate the development of the MV-RNA technology by leveraging our know-how and expertise in LNP delivery as well as our broad understanding of therapeutic RNA payload design,” added Dr. Murray.

Under the license agreement, Tekmira has received exclusive worldwide rights to Halo-Bio’s MV-RNA technology for the development of therapeutic products. Tekmira and Halo-Bio will continue an ongoing research collaboration to design and optimize MV-RNA molecules against gene targets of interest. Financial terms of the license agreement were not disclosed.

The MV-RNA approach to RNAi offers significant advantages over other technologies. Conventional approaches to RNAi utilize double-stranded compounds, known as siRNA, consisting of a guide strand and a so-called passenger strand. Ideally, the guide strand is preferentially incorporated in the RNA induced silencing complex (RISC), where it helps catalyze the site-specific cleavage and subsequent degradation of the targeted messenger RNA. The passenger strand is degraded by cellular nucleases. The MV-RNA technology improves on these aspects of the conventional approach as each strand of an MV-RNA molecule is designed for efficient RISC incorporation and targeted cleavage of a unique mRNA target sequence. Therefore, MV-RNA technology has the potential to increase the potency and minimize off target effects of RNAi drugs.

RNAi therapeutics have the potential to treat a broad number of human diseases by “silencing” disease-causing genes. The discoverers of RNAi, a gene-silencing mechanism used by all cells, were awarded the 2006 Nobel Prize for Physiology or Medicine. RNAi therapeutics require delivery technology to be effective systemically. LNP technology is one of the most widely used delivery approaches for systemic administration of RNAi therapeutics. Tekmira’s LNP technology (formerly referred to as stable nucleic acid-lipid particles or SNALP) encapsulates RNA molecules with high efficiency in uniform lipid nanoparticles, which are effective in delivering RNAi therapeutics to disease sites in numerous preclinical models. Tekmira’s LNP formulations are manufactured by a proprietary method, which is robust, scalable, and highly reproducible, and LNP-based products have been reviewed by multiple FDA divisions for use in clinical trials. LNP formulations comprise several lipid components that can be adjusted to suit the specific application.