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nexiguran ziclumeran (Intellia Therapeutics)
- Development stage
- Phase 3
- Lead developer
- Intellia Therapeutics
- Modality
- Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, mRNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems, Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies
- Administration
- Intravenous
01
Overview
Nexiguran ziclumeran is an investigational, single-administration **in vivo CRISPR-Cas9 gene-editing therapy** for transthyretin amyloidosis. It is a hepatotropic lipid-nanoparticle product containing nexiguran, a TTR-targeting single-guide RNA, and ziclumeran, an mRNA encoding Cas9 endonuclease. Following intravenous administration, the editing system is delivered to hepatocytes and produces durable disruption of the **transthyretin gene**, thereby reducing hepatic synthesis of circulating TTR protein. Intellia Therapeutics leads development and commercialization under its collaboration with Regeneron. The program is in Phase 3 development for ATTR cardiomyopathy and hereditary ATTR amyloidosis with polyneuropathy. ([intelliatx.com](https://www.intelliatx.com/pipeline/clinical-trials/))
- Other names
- nex-z
02
Targets
TTR (Transthyretin)
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