Gene Editing for Hereditary Transthyretin Amyloidosis with Polyneuropathy

Hereditary transthyretin amyloidosis (ATTR) is associated with a rapidly progressive and disabling sensorimotor polyneuropathy (ATTRv-PN) caused by deposition of misfolded transthyretin (TTR) in peripheral nerves. Within the past decade, TTR stabilizers and silencers have transformed ATTR therapy, but most treated patients have stable disease rather than improvement, and many continue to have disease progression. Here, researchers tested whether a single infusion of an investigational CRISPR-Cas9 gene editor — nexiguran ziclumeran (nex-z) — can silence TTR production at its source. The agent is designed for hepatocyte uptake after intravenous delivery, permanently disrupting the TTR gene. In this single-arm, open-label, industry-spon…