clinical / sector
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The Medicine Maker · United Kingdom · Intellia files first in-vivo CRISPR therapy with the FDA
Reports the double-blind HAELO trial as the confirmation regulators need to approve the first in-vivo CRISPR treatment, with the NEJM manuscript published simultaneously to the June EAACI Istanbul presentation; positions it as a category-defining single-dose edit.
“A one-time in-vivo CRISPR therapy has significantly reduced attacks in patients with hereditary angioedema in a phase 3 trial.”