Trying to wrap my head around the potential re-administration of AAV with chimeric AAV | SGMO Message Board Posts

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Msg  128481 of 138255  at  6/19/2019 2:55:31 PM  by

gg100


 In response to msg 128471 by  dnmun
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Re: Trying to wrap my head around the potential re-administration of AAV with chimeric AAV

 agree with dnmun. Sandy didn't say that, or at least if he did he misspoke somehow.
 
If true, Biomarin would be repeating this mantra over and over in every fifth sentence of every  PR or presentation that they make. But Biomarin is completely silent as to redosing except for Worldwide R&D VP Fuchs' claim that they just discovered a new miracle AAV that will re-dose.
 
Re-dosing is the be-all and end-all of genomic medicine because 1) it permits titration of the therapy to fine-tune to the desired level of therapeutic molecule, and 2) when the therapy wanes due to normal cell turnover after 3-10 years (depending on the product, wink-wink) then you just dose again.
 
Maybe Sandy is referring to some complicated method of re-dosing like apheresis to remove all the neutralizing antibodies. This sort of thing can be done, but its no fun for the patient as they have to sit with the aperhesis machine for something like eight hours. It would take some development to do this. 
 
While I think dnmun's thesis that Sangamo grabbed TxCell for the purpose of re-dosing AAV therapies is possible, I do not think it is likely. As Boisell said about a year before SGMO acquired TxCell, all of the oncology cell therapy workers thought the same thing, at about the same time,  "Why not do this with Tregs and attack autoimmune diseases?"
 
So they all observed the radical success of early CD19 CART and thought the same was achievable in autoimmune.
 
The question now is: How is SGMO spending R&D on Treg AAV re-dosing? Is SGMO just testing the waters, or as dnmun has suggested, is Treg AAV re-dosing a major project?
 
And while dnmun may be spot on correct that Fontenot was hired to push Treg AAV re-dosing, I think its more likely that Fontenot was hired to beef up Treg generally. 


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Msg # Subject Author Recs Date Posted
128489 Re: Trying to wrap my head around the potential re-administration of AAV with chimeric AAV dnmun 4 6/19/2019 4:39:22 PM






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