Post by holdencf on Mar 6, 2016 13:51:49 GMT -5
www.alnylam.com/web/assets/ALN-HBV_AASLD_111515.pdf
marcpucket referenced slide 9 as "evidence" of capitulation.
I can't figure out why they are not yet in the clinic because from this AASLD presentation it looks like they have a great candidate. That said, nobody has placed their hand on a bible and stated that they will tell the truth the whole truth and nothing but the truth at AASLD. I don't know enough about the science to know what they may have "accidentally omitted." The one thing that does not pass the smell test is that they reference their LNPs and say that they have a strong safety profile. That may be the case now but it sure as heck was not in the past. I have said it before and I will say it again. I have spoken to both chemists and molecular biologists. Under 100nm these things begin to destabilize. Above 100nm they are too big to achieve an effective amount of cellular uptake. So it's a balancing act. Chop the LNP down to size and whatever bonds that hold them together become unstable or there aren't enough of them left to do the job. When they become unstable they drop their payload too early which creates toxicity. Maybe they came up with some new chemistry in the ESC platform. They certainly have not yet shown as much in humans.
marcpucket referenced slide 9 as "evidence" of capitulation.
I can't figure out why they are not yet in the clinic because from this AASLD presentation it looks like they have a great candidate. That said, nobody has placed their hand on a bible and stated that they will tell the truth the whole truth and nothing but the truth at AASLD. I don't know enough about the science to know what they may have "accidentally omitted." The one thing that does not pass the smell test is that they reference their LNPs and say that they have a strong safety profile. That may be the case now but it sure as heck was not in the past. I have said it before and I will say it again. I have spoken to both chemists and molecular biologists. Under 100nm these things begin to destabilize. Above 100nm they are too big to achieve an effective amount of cellular uptake. So it's a balancing act. Chop the LNP down to size and whatever bonds that hold them together become unstable or there aren't enough of them left to do the job. When they become unstable they drop their payload too early which creates toxicity. Maybe they came up with some new chemistry in the ESC platform. They certainly have not yet shown as much in humans.