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- ATTRibute-CM, BridgeBio’s Phase 3 clinical trial of acoramidis in patients with ATTR-CM, achieved statistical significance in reducing the risk of ACM or first CVH versus placebo in ATTRv-CM patients (59.1% risk reduction), establishing the mechanistic hypothesis that stabilizing TTR may delay or prevent ATTRv-CM

- ACT-EARLY is a registrational, randomized, double blind, placebo controlled, event driven prevention study that will enroll ~600 asymptomatic carriers of a pathogenic TTR variant. Diagnosis of ATTRv disease will be evaluated as the primary analysis of the study

CAMBRIDGE, Mass., March 26, 2025 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies, today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to nexiguran ziclumeran (nex-z, also known as NTLA-2001) for the treatment of transthyretin (ATTR) amyloidosis with cardiomyopathy (ATTR-CM).

Mar 20, 2025

− Novel Mechanism of Action Delivers Rapid Knockdown of Transthyretin, Addressing the Disease at its Source –

− Proven Consistency of Effect on Cardiovascular Outcomes, Function, and Quality of Life in ATTR-CM Population Representative of Today’s Patients –

– Only Therapeutic Approved in the U.S. to Address Both Cardiomyopathy and Polyneuropathy Manifestations of ATTR Amyloidosis −

– Alnylam Offers Multiple Programs to Support Broad and Seamless Patient Access; Majority of Patients Expected to Pay $0 in Out-of-Pocket Costs for AMVUTTRA –

Mystery Solved: New Research Reveals Why a Key Protein Turns Deadly in Aging Bodies
 

By Scripps Research Institute

January 30, 2025

These insights could drive the development of new drugs to treat transthyretin amyloidosis, a progressive and fatal disease.