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."
Labels: SomaGenics, sshRNA, Tekmira