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Re: ghmm post# 223950

Saturday, 03/23/2019 11:38:06 PM

Saturday, March 23, 2019 11:38:06 PM

Post# of 252218

ABEO has a library of > 100 vectors

How do you define vector?

I'd like a clearer explanation of what you mean by vector in this case.

From the Biocentury link:
https://www.biocentury.com/biocentury/regulation/2019-03-08/fda-commissioner-scott-gottlieb’s-exit-interview

FDA thinks this makes sense scientifically because “if you have the same insert in two different expression constructs, you could have very different levels of expression, different clinical effects, different locations of expression, and fundamentally different safety profiles,” Gottlieb said. “We already know this from what we’ve seen to date. Vectors can have a big impact on the overall features of the gene therapy even if the genes are the same.


Typically, and in this case, the "vector" is the delivery package used to deliver a particular gene sequence to the targeted site.

When you say a company has >100 vectors, I assume you mean they used one (or maybe two) vector to package any one of a hundred different gene sequences, not that they have >100 different ways to deliver a particular gene sequence into a cell or organ.

If their approach to gene delivery doesn't work well, or isn't protected by patents, it isn't worth much.

Beware of comparing apples to oranges.

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