Clash of the titans.

Last week we touched on the rapid advancements in gene editing. Well, it was more rapid than we thought. Yesterday the FDA approved a gene therapy for the first time (for any disease) in U.S. history. This is making huge headlines nationwide, though really we’ve known about CAR-T cells (i.e., “gene therapy”) for years and that FDA approval has been in the pipeline for months. So as you’ve probably already heard, tisagenlecleucel (which thankfully also answers to its pop star trade name Kymriah) is now officially an option for the treatment of relapsed or second-refractory ALL in patients ≤ 25 years. More specifically, it is a $475K “option” (...did someone say stock options?) for those with major resources facing a major illness. The real page turner is how this war of worlds will play out among big pharma, with Novartis (with rights to Kymriah) up against arch nemesis KITE (with rights to competitor drug) who was recently bought out by Gilead for a smooth $12 billion….who will all end up in one epic battle after another with CMS and private insurers. Maybe the real blockbuster question is: Does anyone have the movie rights to this yet?

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