RNA strand illustration (Vossman, Wikimedia Commons, via Flickr)
30 July 2018. A biotechnology company is developing treatments for a severe form of hepatitis, which up to now has only limited therapy options, with a synthetic form of RNA designed to shut down the infecting virus. SomaGenics Inc. in Santa Cruz, California is the recipient of a 3-year, $2.9 million award from National Institute of Allergy and Infectious Diseases, part of National Institutes of Health.
SomaGenics is applying its technology using RNA interference to design a treatment for hepatitis D, also known as hepatitis delta virus. Hepatitis D is contracted by people already infected with the more common hepatitis B, and is considered the most severe form of hepatitis in humans. The hepatitis D infectious virus consists of a circular RNA genome surrounded by a coat of hepatitis B envelope proteins. Once infected, hepatitis D can cause severe liver damage leading to end-stage liver disease, liver cancer, cirrhosis, and liver failure. The organization Hepatitis Delta Connect estimates 15 to 20 million people worldwide have hepatitis D.
SomaGenics says alpha interferon, given mainly as a cancer drug, is the only known treatment for hepatitis D, with limited effectiveness in a majority of cases. The configuration of the hepatitis D virus, with its circular RNA genome, makes it a challenging target for most drugs, says the company, thus SomaGenics’ RNA interference could provide a way to treat the disease. RNA interference is a natural process, where RNA molecules limit the expression of genes, also known as gene silencing, an approach being tried for targets such as viruses considered too difficult to address with conventional small molecule drugs.
The SomaGenics technology extends natural RNA interference molecules into what it calls synthetic short hairpin RNAs, or sshRNAs. The company says its sshRNAs are chemically simpler, which means their production and purification is also simpler, and more likely to hit their targets, resulting in fewer adverse effects. Because they are synthetic molecules, the company adds, their dosage and activity life in the body can be better controlled.
In a preliminary project, SomaGenics says it identified inhibitors of hepatitis D, and using the company’s sshRNA platform targeted those inhibitors to stop replication of the virus in lab cultures. The new project aims to develop sshRNAs that can be delivered into the liver, with the aid of lipid, or natural oil, nanoscale particles. The company plans to test a number of different formulations with genetically modified mice that express human hepatitis D. By the end of the project, SomaGenics expects to to have a collection of sshRNAs optimized for efficacy and that minimize resistance, which the company can propose in an Investigational New Drug application to FDA. If approved, that application would allow for the start of clinical trials.
The SomaGenics award was made under NIH’s Small Business Innovation Research program that sets aside funding for small, early-stage businesses in the health care and life science fields. In the current (2018) fiscal year, NIH expects to channel more than $1 billion into its small business set-aside programs.
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