Tekmira Presents Recent Advances in mRNA Delivery

Tekmira Pharmaceuticals Corporation recently announced that recent advances in the enablement of messenger RNA (mRNA) using Tekmira’s lipid nanoparticle (LNP) technology were presented by Tekmira’s Chief Technical Officer, Dr. Ian MacLachlan, at the AsiaTIDES Conference in Tokyo, Japan, from February 25-27, 2014.

“We are pleased to present recent advances with Tekmira’s LNP formulations tailored specifically for mRNA delivery. Our LNP technology is the most widely adopted delivery solution for therapeutics based on RNA interference triggers. However, in addition to RNAi, other important nucleic acid payloads, including messenger RNA, can be efficiently and effectively delivered using Tekmira’s LNP. By leveraging improvements in LNP technology garnered through Tekmira’s siRNA-based product development programs, we have made substantial improvements in mRNA delivery, positioning us to enable the development of mRNA therapeutics by our partners as we have done with RNAi-based therapies,” said Dr. Mark J. Murray, Tekmira’s President and CEO.

This data package builds upon the presentation given at the First International mRNA Health Conference in Tubingen, Germany, in October 2013. In this most recent presentation, titled Recent Advances in the Lipid Nanoparticle-Mediated Delivery of Messenger RNA, data were presented showing highly potent delivery in vivo to liver, tumors, and other tissues. Some key summary points from the presentation include:

• Tekmira’s expertise in LNP delivery is broad and wide-reaching; Tekmira scientists have designed LNP for hepatic delivery, oncology applications, inhalation, and delivery to immune cells.
• Tekmira’s proprietary manufacturing methodology is the method of choice for mRNA delivery and is believed to be the only method resulting in highly reproducible mRNA encapsulation under conditions suitable for product development purposes. Typically, regulatory authorities require greater than 90% encapsulation for GMP and product development purposes, which is a target that is readily achieved using Tekmira’s inherently scalable process.
• Tekmira has successfully integrated third-generation LNP technology in LNP containing mRNA. The result is a more robust manufacturing process, increased encapsulation efficiency, and a substantial increase in mRNA potency relative to second-generation systems, including those that contain MC3.
• Tekmira has demonstrated highly efficient delivery in a range of tumor models – including distal tumors and orthotopic liver tumors – with gene expression long-lived when compared to other tissues.
• When applying Tekmira’s third-generation LNP technology to the delivery of mRNA in the liver, data demonstrated significant improvements over previous generations, readily achieving mRNA delivery and expression in every single hepatocyte.

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, such as siRNAs, require delivery technology to be effective systemically. Tekmira believes its LNP technology represents the most widely adopted delivery technology for the systemic delivery of RNAi therapeutics. Tekmira’s LNP platform is being utilized in multiple clinical trials by both Tekmira and its partners. Tekmira’s LNP technology (formerly referred to as stable nucleic acid-lipid particles or SNALP) encapsulates siRNAs with high efficiency in uniform lipid nanoparticles that 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.

Tekmira Pharmaceuticals Corporation is a biopharmaceutical company focused on advancing novel RNAi therapeutics and providing its leading lipid nanoparticle delivery technology to pharmaceutical partners. Tekmira has been working in the field of nucleic acid delivery for over a decade and has broad intellectual property covering LNPs. Further information about Tekmira can be found at www.tekmirapharm.com.