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Re: iwfal post# 186852

Saturday, 01/31/2015 11:29:33 AM

Saturday, January 31, 2015 11:29:33 AM

Post# of 251654
AGTC

Perhaps because they've got some failures? E.g. They have (had?) an experimental treatment for Leber's Congenital Amaurosis listed in Clinicaltrials that appears to use the same AAV vector they plan to use going forward. Started in 2007 and radio silent. (Note that there were competing treatments that used similar tech and some had some (limited) success - seems to depend upon where you inject it?).

And the non-eye trial they list on their presentation is furthest along (in an on-going ph2b per their presentation), but the data on clinicaltrials says it was essentially a washout. So not sure why it is in ph2b. BTW - I realize that they are now pursuing eyes only, but suggest that dragging along a washout isn't encouraging
.

Another key question is why Genzyme walked away from AGTC and their tech before. Does that raise questions in general about their tech and pipeline or was it just something specific to one drug and a move related in part to Sanofi/REGN's close ties?

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