TTR stabilizers, gene silencing and gene scissors: where do we stand?

Advances in the understanding of the pathophysiology of transthyretin amyloid cardiomyopathy (ATTR-CM) have revolutionized the therapeutic landscape in recent years. TTR stabilizers such as tafamidis and acoramidis can reduce TTR instability and subsequent amyloid fibril formation, and gene silencing therapies with small interfering RNAs such as patisiran and vutrisiran or antisense oligonucleotide inhibitors such as inotersen and eplontersen also represent promising therapeutic options as they reduce TTR production. Further gene silencers, gene therapies and combined treatment strategies are being researched in ongoing studies.