Tekmira Pharmaceuticals Corporation, a leading developer of RNA interference (RNAi) therapeutics, announced that data demonstrating its ongoing lipid nanoparticle (LNP) technology innovations, including the enablement of messenger RNA (mRNA), were presented at the 1st International mRNA Health Conference taking place in Tubingen, Germany from October 23-24, 2013 by Tekmira's Chief Scientific Officer, Dr. Ian MacLachlan.
"Tekmira's LNP technology represents the most widely adopted delivery technology in RNAi, enabling eight clinical trials and administered to over 200 patients to date. Because LNP can enable a wide variety of nucleic acid payloads, including messenger RNA, we continue to see new product development and partnering opportunities based on our industry-leading delivery expertise," said Dr. Mark J. Murray, Tekmira's President and CEO.
"We believe that mRNA represents a class of nucleic acids with exciting therapeutic applications, particularly in rare diseases; however, effective delivery of mRNA is going to be essential for success. We are excited to be presenting data for the first time demonstrating Tekmira's progress with both liver-directed and tumor- directed LNP formulations that are tailored specifically for mRNA delivery," added Dr. Murray.
In a presentation entitled "Lipid Nanoparticle-Mediated Delivery of Messenger RNA" data were presented that showed highly potent delivery in vivo to liver, tumors and other tissues. Some key highlights from the presentation include:
The data demonstrated that mRNA when encapsulated and delivered using Tekmira's LNP technology can be effectively delivered and expressed in liver, tumors and other specific tissues of therapeutic interest.
In one study using mRNA with a liver-directed LNP formulation, data showed gene expression levels orders of magnitude greater than other published results with lipid-based delivery technology.
Tekmira has demonstrated highly efficient delivery in two different tumor models – examining both distal tumors and orthotopic liver tumors – with gene expression long-lived as compared to other tissues.
When applying Tekmira's LNP technology to the delivery of mRNA in the liver, data demonstrated mRNA delivery and expression in every single hepatocyte.
About Messenger RNA (mRNA) and the 1st International mRNA Health Conference
Messenger RNA (mRNA) is a class of RNA molecules that contains the genetic information specifying the amino acid sequence of proteins. mRNA is translated in the cell into polymeric chains of amino acids or proteins. The medical use of mRNA to direct the intracellular expression of therapeutic proteins is rapidly evolving into a key area of research and development in biotechnology. Scientists and pharmaceutical companies around the world are developing drugs based on this biomolecule. The first clinical studies are currently ongoing, and innovative therapeutic treatment options for numerous diseases such as cancer, cardiovascular diseases or influenza are becoming more feasible. The 1st International mRNA Health Conference, taking place on Oct. 23-24, will host the world's experts in mRNA from academia and industry, who will present their latest findings to an expert audience. More information can be found at: www.mrna-conference.com.
About RNAi and Tekmira's LNP
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. Tekmira is based in Vancouver, B.C.