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DewDiligence

03/17/16 12:25 PM

#200301 RE: ghmm #200300

BLUE says re-treatment is “not in their plans.” AGTC says the issue needs further investigation. Not sure what the other GT have said on this subject.
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ghmm

08/19/16 12:31 PM

#203646 RE: ghmm #200300

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?