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Biotech / Medical : ARIAD Pharmaceuticals
ARIA 23.990.0%Feb 17 4:00 PM EST
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To: biowa who wrote (988)2/4/2000 2:24:00 PM
From: Pseudo Biologist  Read Replies (1) of 4474
 
is how will a patient go about using RAPID to supply their insulin needs?

(1) insert appropriate gene in patient (AAV or perhaps ex-vivo viral vector followed by putting cells back in patient). This may have to be done only once in the life, or, most likely, every few years or so?

(2) give patient pills ("anti-dimerizer") to take x times a day

This is what they did for the diabetic mice in the Science paper.

Point 2 requires you have a safe, orally available molecule. This is not too far fetched as the molecule used here may be a very close cousin of what they plan to use for GvHD-related gene therapy. That molecule itself did OK in phase I, showing acceptable oral absorption, good safety, etc.

Hope this is not too stupid an answer to your question -g-

PB
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