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Re: ghmm post# 200300

Friday, 08/19/2016 12:31:07 PM

Friday, August 19, 2016 12:31:07 PM

Post# of 252499
Gene Therapy:

Just to add a bit more about some of the current thinking on retreatment a couple more takes from companies. Take it for what its worth perhaps some with the more modest expression are thinking retreatment and those with higher are not?
DMTX - Think retreatment will be needed but they are not working on it. Think in the next 5 years or so it will be clearer and they plan to be a "follower".
QURE - Think durability may be in the 10 year range. The gene cassette (but different AAV vector) they are using is actually the same as what was used in St. Jude's Hemophilia trial with published results out over 5 years (20l4 published results in NEJM)
http://www.nejm.org/doi/full/10.1056/NEJMoa1407309?af=R&rss=currentIssue#t=article
Uniqure is actually working on retreatment have some research underway and they have a couple posters on their website
http://www.uniqure.com/uploads/Publications/Readministration%20two-step%20AAV%20injection%20ASGCT%20poster2.pdf
http://www.uniqure.com/uploads/Publications/AAV1-AAV5%20readministration%20ASGCT%20poster.pdf

Maybe someone with a better science background could answer something for me. I was wondering if someone treated with one AAV vector would likely develop antibodies that would prevent a different AAV vector from working? If not wouldn't the retreatment option present a nice opportunity for "inferior" regimens of a different type?

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