SI
SI
discoversearch

We've detected that you're using an ad content blocking browser plug-in or feature. Ads provide a critical source of revenue to the continued operation of Silicon Investor.  We ask that you disable ad blocking while on Silicon Investor in the best interests of our community.  If you are not using an ad blocker but are still receiving this message, make sure your browser's tracking protection is set to the 'standard' level.
Biotech / Medical : ARIAD Pharmaceuticals -- Ignore unavailable to you. Want to Upgrade?


To: biowa who wrote (988)2/4/2000 2:24:00 PM
From: Pseudo Biologist  Read Replies (1) | Respond to 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



To: biowa who wrote (988)2/4/2000 3:17:00 PM
From: scott_jiminez  Respond to of 4474
 
The last paragraph of the abstract of the Science paper reads,

This approach may make gene therapy a viable method for delivery of polypeptides that require rapid and regulated delivery.

Following a brief glance at the paper, it would appear the confidence of the authors is supportable.

Back with more detailed impressions later.