SANTA CRUZ, Calif., Sept. 20, 2011 — SomaGenics, Inc., a biotechnology company specializing in RNA-based technologies, presented exciting data on the efficacy of RNA interference (RNAi) therapeutics against Hepatitis C virus (HCV) based on the company's sshRNA platform.
SomaGenics, along with its collaborators at Tekmira Pharmaceuticals and Roche, presented the results in a poster entitled "Synthetic Short shRNAs are Potent Inhibitors of Hepatitis C Virus in HCV-Infected Chimeric Mice" at the 7th Annual Meeting of the Oligonucleotide Therapeutics Society held September 8-10, 2011 in Copenhagen, Denmark.
"The demonstration of a strong antiviral effect in the chimeric human liver mouse model further reinforces our confidence in the therapeutic potential of our sshRNA platform. We are quite excited about the performance of SomaGenics' synthetic sshRNAs formulated with Tekmira's lipid nanoparticles (LNPs), which demonstrated reduction of circulating HCV load by more than two orders of magnitude. The results presented affirm the advantages of our approach to combine multiple sshRNA targeting otherwise 'undruggable' viral targets," said Dr. Brian Johnston, Chief Executive of SomaGenics.
Some conclusions of the scientific presentation:
- This represents the first demonstration of in vivo efficacy of RNAi against HCV infection;
- LNP-formulated sshRNAs were efficiently taken up by human liver cells in a chimeric mouse model;
- Significant HCV reduction (2.0 log10 viral load reduction) was achieved with a single i.v. dose;
- Viral load was still depressed by 1.0 log10 three weeks after dosing;
- Maximal anti-viral effect was observed with two doses of a cocktail of two sshRNAs targeting separate sites on the HCV genome, where a total viral load reduction of 2.5 log10 was achieved.
- The LNP-formulated sshRNAs were well tolerated with no evidence of liver toxicity; and
- The therapeutic effect was convincingly demonstrated to act through an RNAi mechanism.
About Hepatitis C Virus
HCV is a leading cause of cirrhosis, liver cancer, and liver failure requiring liver transplant. An estimated 170 million people are infected with HCV globally, with 3-4 million new infections each year. No vaccine for HCV is currently available.
About RNAi and SomaGenics' sshRNA Technology
The Nobel-prize-winning discovery of RNA interference (RNAi), a process by which double-stranded RNA molecules can inhibit the function of virtually any gene, has spurred interest in the development of short interfering RNAs as drugs. SomaGenics has pioneered the discovery and development of a unique class of short interfering RNAs called sshRNAs. Structurally distinct from the more commonly used siRNAs and expressed shRNAs, sshRNAs are short, chemically modified RNA sequences that are chemically synthesized as single strands. They have outstanding potency and possess attractive pharmacokinetic properties without undesirable immune stimulation.
SomaGenics is a privately held company that develops innovative RNA technologies for therapeutic and diagnostic uses. SomaGenics' lead therapeutic program, against hepatitis C virus, has successfully completed efficacy evaluations in preclinical animal models and is ready for clinical development.
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