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Re: mcbio post# 189829

Saturday, 04/11/2015 11:40:56 AM

Saturday, April 11, 2015 11:40:56 AM

Post# of 252358
QURE:

Sorry if I missed it, but are you generally more optimistic on what BMY aims to do in collaboration with QURE on the gene therapy cardio front or are you as skeptical on that approach as you are with what CLDN is doing?



For me, the major drawback in these programs is the AAV2/x vector's ability to transfect the heart of larger animals. These AAVs invariably show good transfection results when administered by IV in small rodents, but that falls apart in larger animals like dogs, sheep and pigs. Therefore, regardless of the target, I'm interested in the transfection efficiency first and foremost. I've not seen heart data from QURE yet, but would be interested to look if you know of any.

As for the targets, I don't think highly of CLDN's SERCA2a choice. I do think some of the targets that BMY will aim for, such as the congenital cardiomyopathy mutations, are worthwhile. But right now, these are secondary considerations for me.

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