Intellia Therapeutics
Clinical trials sponsored by Intellia Therapeutics, explained in plain language.
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Gene-Editing shot aims to stop rare heart disease in its tracks
Disease control Recruiting nowThis study tests a single dose of NTLA-2001, a gene-editing therapy, in 1,200 adults with a heart condition caused by abnormal protein buildup (ATTR-CM). The goal is to see if it reduces deaths and heart-related events compared to a placebo. Participants must have stable heart fa…
Phase: PHASE3 • Sponsor: Intellia Therapeutics • Aim: Disease control
Last updated May 17, 2026 13:55 UTC
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Gene-Editing shot aims to halt rare nerve disease in phase 3 trial
Disease control Recruiting nowThis study tests a single dose of NTLA-2001, a gene-editing therapy, in 60 adults with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN), a genetic disease that damages nerves. The goal is to see if the treatment can stop or slow the disease by lowering a harmfu…
Phase: PHASE3 • Sponsor: Intellia Therapeutics • Aim: Disease control
Last updated May 06, 2026 16:02 UTC