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Reducing 'Bad' LDL Cholesterol by Silencing Disease Causing Genes

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All of these companies seek to capture a portion of the huge market that exists for the medical need.

"The initial market opportunity might be the sickest patients, maybe those who have had a heart attack or are at risk of another heart attack," said Deutsche Bank analyst Robyn Karnauskas, who added that the segment represents about 3 million people in the United States.

The New York Times points out that this is one of the hottest races in the industy given that millions of Americans cannot lower their cholesterol sufficiently using statins alone, providing a market that could reach billions of dollars in annual sales for a successful drug.

"Nobody knows their sales potential," Karnauskas said, but speculated the class of drugs could fetch $8 billion to $25 billion a year, depending on pricing and how widely they are prescribed.

"New strategies are needed to dramatically and rapidly reduce LDL-C and prevent acute cardiovascular events that result from the rupture of cholesterol rich plaque when patients are at their most vulnerable," said Daniel J. Rader, M.D., professor of Medicine and chief, Division of Translational Medicine and Human Genetics, at the Perelman School of Medicine at the University of Pennsylvania.

Tekmira released their own forward-looking news on Monday, announcing they will delivering data for their lead internal asset TKM-PLK1 at the American Academy of Clinical Research (AACR). Tekmira will deliver Phase I solid tumor data for PLK1 in a podium presentation at the AACR conference. The company previously announced that it has seen positive signs of drug activity in the Phase I study. Clearly Tekmira likes the data they have seen to date, as they are guiding that a Phase II study will be initiated in the second half of 2013.

The study investigators are set to deliver a presentation entitled "A phase 1 dose escalation study of TKM-080301, a RNAi therapeutic directed against PLK1, in patients with advanced solid tumors" and discuss results from Tekmira's Phase 1 clinical trial with TKM-PLK1, which employs a unique lipid nanoparticle developed for oncology applications.

All of these 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.

Their LNP platform has been recognized as the new "gold standard" and is being utilized in multiple clinical trials by both Tekmira and its partners. Investors note that this should ultimately benefit the company, whose shares have been steadily appreciating in value since July of 2012. Observers continue to anticipate that more funds and smart money investors will find their way into these stocks in the days and months ahead, presenting the opportunity for shares to continue to hammer out new lows as they trade across some of recent levels.

The full version of this report can be found here:
http://www.biomedreports.com/20130204121554/reducing-bad-ldl-cholesterol-by-silencing-disease-causing-genes.html

Healthcare investors and Biotech traders interested in accessing BioMedReports' new complete database of clinical trials and upcoming FDA and world-wide regulatory decisions which can be used to make more profitable trades and see upcoming catalysts can go to: http://biomedreports.com/fdacal.html

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Source: Marketwire


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