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Re: Pre_Clinical post# 4254

Monday, 07/23/2007 4:23:09 PM

Monday, July 23, 2007 4:23:09 PM

Post# of 19309
>If this technology is going to be limited to acute indications because the expressed proteins are underglycosylated, and hence more rapidly cleared, then that is something I would like to know about.<

There’s no evidence of this. As I said in the previous post, the isoform differences between ATryn and endogenous AT were the luck of the draw, and this information comes directly from Dr. Meade. Thus, there is no reason to assume that other GTC drug candidates will have the same kinds of alterations to the glycosylation that Atryn does.

Moreover, you make the assumption that under-glycosylation necessarily implies a shorter half-life. What is your basis for saying this?

Finally, let me point out that GTC’s FVIIa and AAT programs are intended to address medical indications where chronic therapy is needed. This in itself would seem to refute your suggestion that GTC’s platform is only suited to making drugs for acute indications.

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