Benitec Biopharma (ASX: BLT) is investigating long term gene silencing using DNA-directed RNA interference (ddRNAi) for human therapeutics. The company’s primary therapeutic program focuses on human immunodeficiency virus (HIV). Its other projects are in the area of infectious diseases and cancer.
Benitec Biopharma (ASX: BLT) is progressing towards human clinical studies with the company’s multiple gene silencing technology programs.
Trials of Benitec’s HIV/AIDS program have showed that the ddRNAi construct still expresses 3.5 years after the patient’s stem cells were transfected with a lentivirus construct.
Benitec licensee Calimmune is targeting a clinical trial in HIV/AIDS patients in 2012, the first trial to use only ddRNAi constructs to target HIV in patients.
In the United States, Benitec subsidiary Tacere Therapeutics has progressed the company’s Hepatitis C program to the point where an Investigational New Drug application is being prepared.
Discussions with the US Food and Drug Administration are advanced.
Already a large amount of clinical trial data has been manufactured, with a U.S. based clinical trial to begin as early as 2013.
Meanwhile, pre-clinical testing of lead compounds for use in treating cancer-associated pain has been simplified, with all test species confirming strong activity against the target gene.
Production of a material suitable for pre-clinical and clinical trials is now underway.
Testing of Benitec’s other programs, which target areas such as non-small cell lung cancer, hepatitis B and oculopharyngeal muscular dystrophy, are also progressing well.
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