Interesting article on GT and pharma, page-12

J8, the shrna numbers don't tell us if knockdown is occurring. If knockdown is occurring, they don't tell us the extent of that knockdown. It would be wrong to assume that, because we have shrna's produced at levels seen as therapeutic in pre-clinical studies, these translate directly into therapeutic levels in humans. So far, only replicon model data is available about the shrna levels and knockdown. We need to know that the trails have achieved similar knockdown outcomes. So many trials fail because pre-clinical success does not translate into clinical outcomes. A confirmation of the replicon model would be almost as good as SVR data.