SomaGenics Reports on successful HCV virus inhibition with its therapeutic sshRNA.

SANTA CRUZ, Calif., Jan 06, 2014  SomaGenics published exciting data on the efficacy of RNA interference (RNAi) therapeutics against Hepatitis C virus (HCV) based on the company’s sshRNA platform. Using a mouse model with humanized liver that supports infection by HCV, SomaGenics scientists and their collaborators from Roche and Tekmira Pharmaceuticals demonstrated potent, long-lasting reduction in viral load with a mixture of its lead sshRNAs targeting HCV.

The study, published in Gastroenterology, the premiere journal in the field, represents the first demonstration of in vivo efficacy of a synthetic RNAi agent against HCV infection. Key findings of the paper include:

• LNP-formulated sshRNAs were efficiently taken up by human liver cells in the 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 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.

“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,” said Dr. Brian Johnston, Chief Executive of SomaGenics. “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."

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.

About SomaGenics

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.

The citation for the paper is Ma, Dallas et al., Gastroenterology (2014) 146: 63-66. Aspects of the findings have been presented at the following scientific meetings:

June 2014
Nucleic Acid Research & Discovery conference, GTC, San Diego; Featured lecture by BJ

May 2014
American Society for Gene and Cell Therapy Annual Meeting (ASGCT), Washington DC; poster presentation by BJ

April 2014
Symposium of Viral Hepatitis, Varadero, Cuba; invited talk by BJ

March 2014

• International RNA Summit, UC Sant Cruz; invited talk by BJ

• MicroRNA as Biomarkers and Diagnostics Conference, Boston; invited talk by BJ and BJ moderated a Roundtable on Detection of Circulating microRNAs

• University of Massachusetts Medical School; seminar by BJ

Upcoming presentations

July 2014
3rd International Conference on Gastroenterology & Urology, San Francisco, July 28-30, 2014; invited talk by BJ

November 2014
American Association of Pharmaceutical Scientists (AAPS) Annual Meeting, Workshop on Emerging Trends in Nucleic Acid and Cell-Based Therapeutics, San Diego; invited lecture by BJ