Intellia Therapeutics
Clinical trials sponsored by Intellia Therapeutics, explained in plain language.
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Gene-Editing shot aims to halt rare nerve disease in phase 3 trial
Disease control Recruiting nowThis phase 3 trial tests a single dose of NTLA-2001, a gene-editing therapy, in 60 adults with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN), a rare genetic disease that damages nerves. Participants are randomly assigned to receive either the active treatmen…
Phase: PHASE3 • Sponsor: Intellia Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 12:08 UTC
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Gene-Editing breakthrough: could a single dose stop a deadly heart condition?
Disease control Recruiting nowThis study tests a single dose of NTLA-2001, a gene-editing therapy, in 1200 adults with transthyretin amyloidosis with cardiomyopathy (ATTR-CM), a condition where abnormal protein builds up in the heart. The goal is to see if it reduces heart-related deaths and events compared t…
Phase: PHASE3 • Sponsor: Intellia Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 12:01 UTC