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CRISPR-Cas9

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Cardiology

First-in-Human CRISPR-Cas9 Editing of ANGPTL3 Shows Dose‑Dependent Protein Knockdown with Acceptable Short‑Term Safety in Phase 1 Trial

A phase 1, ascending‑dose study of CTX310 (LNP-delivered CRISPR-Cas9 targeting ANGPTL3) in 15 patients produced dose-dependent ANGPTL3 reductions at ≥0.6 mg/kg with few acute safety signals; longer follow‑up and larger trials are required t

Nov 10, 2025
Clinical Updates

Nexiguran Ziclumeran Gene Editing: A Pioneering Treatment for Hereditary ATTR with Polyneuropathy

Nexiguran ziclumeran, an innovative CRISPR-Cas9 gene-editing therapy, demonstrates rapid, sustained suppression of transthyretin (TTR) in hereditary ATTR polyneuropathy patients, showing promising late-phase clinical safety and efficacy.

Sep 26, 2025Sign in to read

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