Thread by @BioCompounding

$NTLA and $REGN report initial data from ph1 study of #ATTR for NTLA-2001. All patients across the two doses showed 92-94% TTR reduction at day 28.
#crispr #genomics #genetherapy #biology #biotech

At both dose levels, $NTLA-2001 was generally well tolerated. 2 patients reported transient infusion reactions, which was the only observed treatment-related adverse event.

$NTLA-2001 is an in-vivo administered #genetherapy.
It is one of the first few in-vivo #GeneTherapy being tested. Congrats to the $NTLA and $REGN teams.

Full details of the update from $NTLA can be found here. #crispr #biotech #GeneTherapy #GeneEditing #biology #genomics #genetics
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