As I have said before, it seems that silence and replace will be where we are heading in the foreseeable future - with the exception of BB-4/501 because of the Nant involvement. The current success of BB-301 may lead us to believe that this is a relatively safe path to take, however, others have tried this approach with different indications only to be thwarted by delivery. Also, some of the monogenetic disorders, such as sickle cell and duchenne muscular dystrophy, have several other biotechs/researchers hotly pursuing them.
It may be smarter for us to take a leaf out of Alnylam's book and concentrate on diseases in cells/tissues that are the easiest to reach with a vector (viral or otherwise). We may have to do more work ourselves on delivery or we could have relationships with others, such as 4D. When we know what the target cells are, we can then work out what diseases to target and the design of the therapeutic.
In Alnylam's case, they quickly worked out that, initially, they could only deliver sirna to the liver and so they directed all their attention to liver diseases and the refinement of delivery to the liver. Look at their market cap and tell me that this was not a smart move on their part.
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