Intellia's CRISPR program that edits genes directly in patients shows durability in ATTR amyloidosis
The first in vivo CRISPR/Cas9 gene editing program has some new durability data showing sustained reduction of a toxic protein in ATTR amyloidosis at all four dose levels in a small 15-patient study.
Intellia Therapeutics presented the much-anticipated data for its Regeneron-collaborated NTLA-2001 Friday morning, adding to the initial Phase I results it first delivered almost a year ago to the day.
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