HORSHAM, PA (January 11, 2008): Nucleonics, Inc., a privately held biotechnology company focused on the development of novel RNA interference (RNAi)-based therapeutics, announced today that the company has begun treating patients in a Phase 1 human safety study of its experimental treatment for chronic Hepatitis B virus (HBV) infection, NUC B1000.
NUC B1000 (http://www.nucleonicsinc.com/products/hepb.html) is an expressed interfering RNA (eiRNA)-based product consisting of a plasmid DNA construct designed to produce four short interfering RNA (siRNA) molecules, formulated with a proprietary cationic-lipid delivery system. Each of the four siRNAs targets a different sequence of the HBV genome, collectively leading to the potential destruction or elimination of all RNA species produced by HBV within an infected cell. The result is a potent antiviral effect designed for efficacy against all HBV genotypes, including drug resistant strains. Additionally, unlike currently available HBV therapeutics, NUCB1000 is designed to specifically reduce viral antigen load in addition to viral titer, thereby reducing the destructive effects of hepatitis and increasing the potential for resolution of viral infection.
Despite the widespread availability of multiple prophylactic vaccines against HBV, chronic HBV infection remains a public health problem around the world that can lead to such serious diseases as cirrhosis, fibrosis and liver cancer in up to one-third of patients, said Principal Investigator, Robert G. Gish, M.D, Medical Director, Liver Transplant Program and Chief, Division of Hepatology and Complex GI, California Pacific Medical Center, San Francisco. Current anti-HBV drug therapies are of limited effectiveness and are not often used in patients with mild to moderate disease due to risks of developing drug resistance. Thus there is significant need for new treatments that can improve
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