Will a CRISPR-based gene editing therapy for transthyretin (ATTR) amyloidosis be available in the US by 2027?
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This is the first the first in vivo CRISPR-based candidate to begin late-stage clinical development. Phase 3 trials expected in late 2023. https://www.europeanpharmaceuticalreview.com/news/187777/regulatory-first-for-gene-editing-therapy/
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The current price is approximately the percent of drugs entering Phase III trials that become approved: https://www.science.org/content/blog-post/latest-drug-failure-and-approval-rates
That seems reasonable for now, but I'll definitely watching this closely. Thanks for posting this question, I wouldn't have known about this otherwise!
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