"But attempting to draw the conclusion that manufacturing is stable from the fact that all manufacturing lots have equally wide patient-to-patient seems preposterous to me."
They are trying to convince the FDA is that internal patient factors cause the variability and is not the fault of its drug..
Thus they are willing to show a slide about how crappy their effectiveness is..
Their argument is that every patient is effected by drugs differently, such as coumadin, blood pressure meds, and even chemo, so its not the fault of the drug or its manufacturing...its the patients fault..
Very weak argument if they compare it to current standard of care in Hemophilia or compare to SGMO results.
But since they are first, they may get a "pass" since SGMO drug not filed for approval yet.