Episodes

  • For FSHD gene therapy you need to "Have-a-Little-Heart"
    Oct 11 2024

    "You know the Greeks didn't write obituaries. They only asked one question after a man died: 'Did he have passion?'. " Today we are joined by our CRISPR Goddess to discuss her new FSHD optimized gene therapy cassette that is stronger, safer, more compact and all around better than anything out there for use in FSHD and neuromuscular gene therapy approaches.

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    32 mins
  • Fulcrum follow-up with our Angry Dad
    Sep 28 2024

    "Show me the money!" Brad our Angry Dad sits down with us and asks the questions on his mind about the Fulcrum results and others going forward.

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    1 hr and 37 mins
  • Our take on the REACH losmapimod clinical trial results
    Sep 19 2024

    "I always tell the truth, even when I lie." Today we are joined by our CRISPR Goddess and cover the Fulcrum Therapeutics REACH phase 3 clinical trial data release and discuss our journey with Fulcrum from the beginning. While very disappointing from several aspects, in the end a lot of benefit truly has been gained for the FSHD field and we are grateful for Fulcrum's contributions to help others going forward. And there are many others coming along, this is the beginning and not the end for FSHD therapeutics.

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    1 hr and 58 mins
  • A FORTITIDE follow-up with our (not so) Angry Dad.
    Jun 22 2024

    "There's a time for daring and there's a time for caution, and a wise man understands which is called for." Brad our Angry Dad is back with some questions for us on the Avidity FORTITUDE AOC 1020 phase 1/2 trial interim data report and some muscle building.

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    1 hr and 16 mins
  • A(+) is still for Avidity: give me the good stuff!
    Jun 14 2024

    "Whatchyou talkin' 'bout Willis?" In a field where every press release is met with unsubstantiated hoopla and proclamations of BREAKTHROUGH! to fire you up to shake you down, we finally have some real news. We provide our evaluation of Avidity's public interim report on their FORTITUDE AOC-1020 phase 1/2 trial.

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    1 hr and 6 mins
  • Pigs and p38
    Jun 1 2024

    "You're messing with the wrong guy!!!!" We expand a bit more on the utility of the FSHD-like pig model and then discuss the science behind and implications of p38 inhibition for FSHD.

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    1 hr and 34 mins
  • The three little (FSHD) piggies.
    Jan 28 2024

    "We don't get a lot of things to really care about." So, you think you are interested in science, eh? Well, let's see how the sausage is made. Today we share our experience generating the FSHD-like minipig models, which will be key tools for testing and advancing better FSHD therapeutics and developing methods for building back your muscles.

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    2 hrs and 11 mins
  • CRISPR in the clinic
    Dec 24 2023

    "Come out to the coast, we'll get together, have a few laughs..." The holiday season we discuss the recent FDA approved CRISPR therapy for sickle cell disease and some dynamics of methylation.

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    59 mins