Three Somagenics Publications Demonstrate Successful Inhibition of the Hepatitis C Virus by Therapeutic sshRNA™ and Confirm Mechanism of Action

SANTA CRUZ, Calif., Dec. 19, 2014 /PRNewswire/ — SomaGenics and collaborators have published exciting results on the efficacy of RNA interference (RNAi) therapeutics against the hepatitis C virus (HCV) based on the company's sshRNA™ platform.  Using a chimeric mouse model with humanized liver that supports infection by HCV, SomaGenics scientists and their collaborators from Roche and Tekmira Pharmaceuticals demonstrated efficient sshRNA delivery to the liver, potent and long-lasting reduction in viral load, and strong evidence for a direct anti-viral effect by the sshRNAs.  The studies were published in Molecular Therapy-Nucleic acids, in Gastroenterology, and in the Journal of Virology.

Key findings of these papers include:

• Lipid-nanoparticle (LNP)-formulated sshRNAs were efficiently taken up by human liver cells in chimeric mice
• 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 different 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
• The therapeutic effect was convincingly demonstrated to act through an RNAi mechanism
• The results illustrate the value of a feature of RNAi approaches: the ability to easily target multiple sites on the viral genome simultaneously

"The demonstration of a strong antiviral effect in the chimeric humanized-liver mouse model further reinforces our confidence in the therapeutic potential of our sshRNA platform," said Dr. Brian Johnston, CEO of SomaGenics. "We are quite excited about the performance of SomaGenics' synthetic sshRNAs formulated with Tekmira's LNP, which demonstrated reduction of circulating HCV load by more than two orders of magnitude and suggests that biweekly or monthly dosing could be effective. The results affirm the advantages of our approach of combining multiple sshRNAs targeting otherwise 'undruggable' viral targets.  We are extending this platform to developing treatments for hepatitis delta, a serious form viral hepatitis for there are currently no specific treatments."

About HCV
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 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 and mechanistically distinct from the more commonly used siRNAs and expressed shRNAs, sshRNAs are short, chemically modified RNA sequences that are synthesized as single strands. They have outstanding potency and possess attractive pharmacokinetic properties without undesirable immune stimulation.

About SomaGenics
SomaGenics is a privately held biotech company with offices and laboratories located in Santa Cruz, CA.  It specializes in RNA technologies, including innovative therapeutics and diagnostics that use RNA molecules as therapeutic targets and biomarkers.  Besides sshRNA, its technology platforms include miR-ID™, a novel, circularization-based RT-qPCR method for quantifying microRNAs; miR-Direct™, for detection of circulating microRNAs directly from plasma; miR-ACS™, for construction of improved next-generation sequencing libraries; and mR-FQ™, for quantifying fragmented mRNAs from preserved histology specimens.

The publications

1. A. Dallas, H. Ilves et al., Mol. Therapy–Nucleic Acids (2013): 17;2:e123
2. H. Ma, A. Dallas et al., Gastroenterology (2014) 146: 63-66
3. A. Dallas, H. Ilves et al., J. Virology (2014) 88(9):4647-56

Aspects of the findings have also been presented by Dr. Johnston at the following scientific meetings:

Media Contact:
Brian Johnston
831 426 7700
Email