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Message: ALNY/BTI

Koo,

As far as I know, there is no connection between BTI and ALNY. ALNY is a leader in siRNA technology. Their lead drug patisiran was shown to reduce TTR levels and ameliorate the neuropathy symptoms of hereditary ATTR amyloidosis with polyneuropathy and improve quality of life. These patients make a mutant TTR protein that lead to the amyloidosis. So patisiran reduces the expression of both normal and mutant TTR to reduce amyloidosis. 

TTR is primarily made by the liver and choroid plexus in the brain. However, current siRNA and somewhat similar antisense oligonucleotide technologies don't cross the BBB very efficiently if at all. The effects of patisiran are likely due to the reduction of liver derived TTR.

It is possible that attaching the BiOasis xB3 linker to patisiran would also permit reduction of choroid plexus derived TTR.

So long story short, there is no connection between BTI and ALNY, but what a beautiful connection that would be to connect xB3 to ALYNs siRNAs to get them across the BBB! 

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