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Re: mcbio post# 206100

Wednesday, 11/16/2016 7:47:37 AM

Wednesday, November 16, 2016 7:47:37 AM

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QURE:

The other (pessimistic) view is that they could resort to being a contract manufacturer. They hinted on the call about possible BD in that area. They will need no more than 500L capacity for the foreseeable future and can scale current facility to 2000L.

Of course I wonder why they won't succeed in Hemophilia B (I've discussed these before):
1) Their Vector is different and could potentially serve patients other GT cannot
2) A FIX expression > 10% would still be revolutionary compared to what exists today and would eliminate prophylactic factor replacement which is really the more meaningful clinical endpoint then an arbitrary expression percentage.
3) The company reiterated on the call their durability (6.5+ years now) with the same cassette and no loss expression as seen with other programs. So if ONCE is starting much higher but the expression diminishes over time one may prefer a more consistent albeit lower starting level (remains to be seen since both need much longer followup).

And Still their BMS collaboration remains one of the (if not the) most lucrative GT deal with a relatively novel (less competitive) area of focus.

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