CRISPR-Cas9 Gene Editing with Nexiguran Ziclumeran for ATTR Cardiomyopathy

A phase 1 trial of nexiguran ziclumeran (nex-z) to treat transthyretin cardiomyopathy showed a rapid and durable reduction of 90% in serum transthyretin levels and evidence of disease stabilization.

​   The New England Journal of Medicine: Search Results in Neurology/Neurosurgery