Tekmira Pharmaceuticals Corporation, a leading developer of RNA interference (RNAi) therapeutics, announced that promising preclinical data from its collaboration with the National Cancer Institute (NCI) has been published recently.
The article presents encouraging pre-clinical data related to the treatment of liver cancer using small interfering RNA (siRNA) enabled by Tekmira's lipid nanoparticle (LNP) delivery technology.
"We are pleased to see further exciting CSN5 anti-tumor data coming out of our ongoing collaboration with the National Cancer Institute. We believe that the NCI's expertise in identifying novel cancer targets is complemented by Tekmira's expertise in siRNA molecule design and LNP delivery," said Dr. Mark J. Murray, Tekmira's President and CEO.
Scientists at the NCI have worked in collaboration with Tekmira to develop siRNA to silence the CSN5 gene, which could represent a novel therapeutic target to treat hepatocellular carcinoma (HCC) and other cancers. This latest data indicates that siRNA-mediated CSN5 knock-down inhibits cell-cycle progression and greatly increases the rate of apoptosis (programmed cell death) in HCC cells. Some key highlights from the article include:
- Gene expression data indicating that CSN5 has a pivotal role in HCC pathogenesis and microarray analysis revealing CSN5 may be one of the early markers of malignant conversion of HCC.
- Optimized siRNA against CSN5 resulted in 80% inhibition of tumor cell growth in vitro.
- Systemic delivery of the siRNA using Tekmira's LNP delivery technology resulted in a significant reduction in tumor growth in a model of human liver cancer.
"We look forward to continuing our collaborative work with the NCI to identify novel cancer targets and demonstrate anti-tumor activity by silencing these genes through RNA interference with the goal of advancing new oncology product candidates," added Dr. Murray.
CSN5 over-expression has been observed in a number of cancers, including breast, thyroid, skin, ovarian, lung, and pancreatic cancers. HCC is the third most lethal cancer, causing an estimated 600,000 deaths annually due in part to a lack of effective treatment options.
Tekmira continues to advance its pipeline of proprietary product candidates, including Tekmira's lead oncology product, TKM-PLK1, which is currently in a Phase 1 human clinical trial conducted at three medical centers in the United States.
Illustration: Tekmira Pharmaceuticals Corporation.
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Tekmira Pharmaceuticals Corporation News Release (04/21/11)
Abstract (Oncogene; (04/18/11))