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Posted: Oct 26, 2010

Tekmira and Collaborators at the NCI Publish Promising Preclinical Data Demonstrating Anti-Tumor Activity of a Novel Cancer Target via RNA Interference

(Nanowerk News) Tekmira Pharmaceuticals Corporation, a leader in RNA interference (RNAi) therapeutics, announced that, together with collaborators at the National Cancer Institute (NCI), it has published promising preclinical data in the prominent journal Cancer Research (Lee et al, Definition of Ubiquitination Modulator COP1 as a Novel Therapeutic Target in Human Hepatocellular Carcinoma. Cancer Res; 70(21); 8264-9). The data stem from the use of small interfering RNA (siRNA) enabled by Tekmira's lipid nanoparticle (LNP) technology in preclinical models of human hepatocellular carcinoma (HCC) or liver cancer.
Scientists at the NCI identified a novel oncology target called COP1 that is over-expressed in human liver cancers and can accurately predict patient survival. Tekmira and scientists at the NCI designed specific siRNA that, when delivered systemically with Tekmira's LNP technology (COP1-LNP), silence the COP1 gene. In preclinical studies, COP1-LNP demonstrated a greater than 12-fold decrease in liver cancer cell growth in tumor bearing mice. This tumor growth inhibition exceeds the National Cancer Institutes criteria for promising therapeutic compounds.
Dr. Mark J. Murray, Tekmira's President and CEO, said "This work is an illustration of one approach Tekmira is taking to identify targets of therapeutic interest which could lead to the development of product candidates. We are very encouraged by the results of this study and look forward to advancing this work with our collaborators at the NCI. By combining the NCI's expertise in identifying novel cancer targets and Tekmira's leadership in siRNA molecule design and delivery, we intend to build on what has been a very successful collaboration over the past several years."
COP1 has been identified as being over-expressed in human HCC and a predictor of patient survival. The silencing of COP1 via RNAi affects a number of genes in a cancer cell including restoring the expression of p53, a key tumor suppressor protein. The result is an inhibition of cancer cell proliferation and programmed cell death.
Primary liver cancer or HCC is associated with one of the poorest survival rates in oncology with only 30-40% of patients being eligible for curative treatment due to late diagnosis, underlying liver disease and lack of effective treatment options. HCC is the third most lethal cancer with an estimated 600,000 deaths annually.
About RNAi and Tekmira's LNP Technology
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. LNP technology is one of the most widely used siRNA delivery approaches for systemic administration. Tekmira's LNP technology (formerly referred to as stable nucleic acid-lipid particles or SNALP) encapsulates siRNAs 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.
About Tekmira
Tekmira Pharmaceuticals Corporation is a biopharmaceutical company focused on advancing novel RNAi therapeutics and providing its leading LNP 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 Tekmira is based in Vancouver, B.C.
Source: Tekmira (press release)
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