Positive clinical data from the Phase 1 trial of nexiguran ziclumeran

First clinical evidence from ongoing Phase 1 study that nexiguran ziclumeran (nex-z), an in vivo CRISPR/Cas9-based gene editing therapy, may favorably impact disease progression in transthyretin (ATTR) amyloidosis. Data was published in NEJM and presented at the 2024 American Heart Association Scientific Sessions.