Date: 2015-10-07
Type of information: Development agreement
Compound: ddRNAi-based hepatitis C therapy TT-034
Company: Lonza (Switzerland) Benitec BioPharma (Australia)
Therapeutic area: Infectious diseases
Type agreement: development manufacturing production
Action mechanism: gene therapy. DNA-directed RNA Interference (ddRNAi) is a platform for silencing unwanted genes. Benitec\'s patents cover the use of RNAi resulting from the introduction of a DNA construct to the target human cell nucleus in vivo, to effect long-lasting genetic silencing. ddRNAi introduces a DNA sequence directly into the cell’s nucleus, and to do that, a range of well-characterized gene therapy vectors, viral and non-viral, like lentivirus, adenovirus, AAV or modified polyethylenimine can be used. The DNA sequence codes for specific shRNAs which are processed to siRNAs and complete the RNAi cycle in the cytoplasm. Herein lies another major difference: because of the delivery mechanism and site, only a minute dose (perhaps as few as 5 copies) of the DNA construct is needed, yet the genetic change is long-lasting, because the shRNA continues to be expressed for long periods, potentially up to years from the integrated DNA construct (Sci Trans Med 2010). ddRNAi can also be used to target multiple genes or multiple parts of one gene, so more complex conditions can be treated with tiny doses, without the side-effects of high-dose, long term administration.
Disease: hepatitis C
Details: * On October 7, 2015, Lonza Houston, a global leader in biological, cell and viral gene therapy manufacturing, and Benitec Biopharma, a clinical stage biotechnology company commercializing a patented gene-silencing technology, DNA-directed RNA interference (ddRNAi), announced that they have entered into a Manufacturing Services Agreement to develop a scalable manufacturing process for Benitec’s ddRNAi-based, Adeno-Associated Virus (AAV)-delivered products intended for therapeutic use
in humans.
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