TRANSTHYRETIN-RELATED (ATTR) FAMILIAL AMYLOID CARDIOMYOPATHY

Clinical trials for TRANSTHYRETIN-RELATED (ATTR) FAMILIAL AMYLOID CARDIOMYOPATHY explained in plain language.

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Completed 1

Gene-Editing breakthrough tested for rare, fatal heart and nerve disease

Disease control Completed

This early-stage study tested a new, one-time gene-editing treatment called NTLA-2001 for people with hereditary forms of amyloidosis, a disease where a harmful protein builds up and damages the heart or nerves. The main goal was to check the safety of the treatment and see how i…

Matched conditions: TRANSTHYRETIN-RELATED (ATTR) FAMILIAL AMYLOID CARDIOMYOPATHY

Phase: PHASE1 • Sponsor: Intellia Therapeutics • Aim: Disease control

Last updated Mar 17, 2026 12:55 UTC

TRANSTHYRETIN-RELATED (ATTR) FAMILIAL AMYLOID CARDIOMYOPATHY

Get alerted when new TRANSTHYRETIN-RELATED (ATTR) FAMILIAL AMYLOID CARDIOMYOPATHY trials appear

Gene-Editing breakthrough tested for rare, fatal heart and nerve disease