BMSN DD
Licensed technology: Less toxic, longer lasting. more efficient.
Benitec/BMSN license arrangement-- ddRNAi technology uses a different molecule and mechanism to effect change in a different part of the cell, enabling smaller doses, lower toxicity, longer-lasting effects and wider application than most gene silencing technologies.
RNAi and disease control
The mechanism of RNA interference (RNAi): In the context of gene silencing for disease control, this is how it is done: if you silence a particular gene in a cell, a specific protein won’t be made so, potentially, you can treat, cure or influence the disease or disorder caused by that protein.
How ddRNAi is unique:
In summary, in comparison to other gene silencing methods, the advantages of ddRNAi are:
· Less fragile: construct is based on DNA not RNA, so is more robust and less easily degraded
· Easier delivery: established, well-characterized gene therapy vectors can be used
· Higher efficiency: up to 250 times compared to siRNA, and up to 1000 times compared to antisense, enabling significantly smaller doses to be used
· Persistence: for up to years compared to hours or days, due to genetic change in the nucleus not transient change in the cytoplasm
· Single or few doses: no need for ongoing administration
· Lower toxicity: due to lower dose, short term administration
· Lower immune activation: due to lower dose, short term administration
· Fewer off-target effects: due to lower dose, short term administration
· Wider application: many genes or many parts of one gene can be targeted with one dose. Can be administered ex vivo, or injected locally or systemically.
· Global application: the ddRNAi platform can be licensed for many disease programs.
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