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 1200 adults with transthyretin amyloidosis with cardiomyopathy (ATTR-CM), a condition where abnormal proteins damage the heart. The goal is to see if the treatment reduces deaths and heart-related events comp…
Phase: PHASE3 • Sponsor: Intellia Therapeutics • Aim: Disease control
Last updated May 26, 2026 03:44 UTC
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One-shot gene therapy aims to stop rare nerve disease in its tracks
Disease control Recruiting nowThis study tests a single dose of a gene therapy called NTLA-2001 in 60 people with a rare inherited nerve disease (ATTRv-PN). The goal is to see if it can safely reduce harmful protein buildup and slow nerve damage compared to a placebo. Participants must have a confirmed diagno…
Phase: PHASE3 • Sponsor: Intellia Therapeutics • Aim: Disease control
Last updated May 22, 2026 14:01 UTC