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Advances in the treatment of transthyretin cardiac amyloidosis: Current and emerging therapies

Transthyretin cardiac amyloidosis (ATTR‐CA) has been recognized as an underdiagnosed and undertreated cause of heart failure with often unrecognized multiorgan involvement. Guideline development and the establishment of nonbiopsy criteria for diagnosis of ATTR‐CA have led to an increased rate of diagnosis and hence patients referred for therapies. ATTR is a protein misfolding disorder where the TTR tetramer disassociates into monomers which form insoluble amyloid depositions in organs, including the heart. ATTR‐CA can be due to autosomal dominant transmitted gene mutation or due to misfolding of wild‐type TTR. Prior to 2019, there were no FDA‐approved pharmacological treatments for ATTR‐CA. Understanding of ATTR‐CA pathogenesis has enabled development of targeted strategies with novel disease‐modifying therapies. Current and emerging therapies for ATTR‐CA include (1) TTR gene silencing (siRNA, ASO, CRISPR/Cas9), (2) TTR tetramer stabilization, and (3) TTR amyloid fibril degradation. This review focuses on the pathophysiology of ATTR‐CA, diagnostic criteria, and addresses current and emerging treatments for this diverse disorder.