Transthyretin-related (ATTR) familial amyloid polyneuropathy

Clinical trials for Transthyretin-related (ATTR) familial amyloid polyneuropathy explained in plain language.

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Gene editing breakthrough? CRISPR therapy targets rare amyloidosis

Disease control Completed

This early-stage trial tests NTLA-2001, a CRISPR-based gene editing therapy, in 72 adults with hereditary ATTR amyloidosis—a condition where abnormal protein builds up in nerves or the heart. The treatment aims to turn off the faulty gene to reduce protein levels. The study focus…

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

Phase 1 • Sponsor: Intellia Therapeutics • Aim: Disease control

Last updated Jun 27, 2026 09:00 UTC

Transthyretin-related (ATTR) familial amyloid polyneuropathy

Get alerted when new Transthyretin-related (ATTR) familial amyloid polyneuropathy trials appear

Gene editing breakthrough? CRISPR therapy targets rare amyloidosis