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This is actually really promising. Their approach is currently the safest with the most clinical data behind it (across all their current treatments), especially when you see headlines like this:

"A third patient has died in Audentes’ pivotal gene therapy trial for X-linked myotubular myopathy, adding fresh concerns for the field right on the heels of BioMarin’s surprise slapdown at the FDA.

The biotech, bought out by Astellas just 8 months ago for $3 billion, says the third patient died from gastrointestinal bleeding. And like the two others who died, this person was on the high dose."

I had peaked at Audentes but decided it was still too early, then they got bought out. AAV appears to have significant strings attached that lentiviral vectors / Bluebirds approach does not. Bluebird seems to still be firing on all cylinders, moving forward ide-cel for multiple myeloma, CALD seems to be approved within a year in US & EU, Zynteglo approved, etc.

btw, editas has started treatment in a whole 3 patients thusfar. ::eyeroll emoji::
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