ANP 2.08% 4.7¢ antisense therapeutics limited

Why Did ATL 1102 Fail In Leukemia Study In Vivo?

  1. 6 Posts.
    I've been doing some DD on ANP and came across this study using ATL 1102 in mice both in vitro and in vivo.

    https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5678723/

    The study shows that ATL 1102 successfully down-regulated its target in vitro but NOT in vivo.

    This is making me nervous about the DMD results. Look, on the surface the clinical DMD results look great. But it's a small, uncontrolled study. I'm very worried about the placebo effect. I was an investor in IIL and IIL compassionate use SPMS patients swore up and down IIL was working for them. Then the trial results came out and showed IIL had "no clinically meaningful or statistically significant" results in a controlled study over placebo.

    What's to say that ATL 1102 in a Phase 2B study doesn't turn out exactly like IIL or the ATL 1102 Leukemia study?
 
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