nexiguran ziclumeran (nex-z, NTLA-2001)
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Indications
- nexiguran ziclumeran is an investigational therapy based on CRISPR-Cas9 (clustered regularly interspaced short palindromic repeats & associated Cas9 endonuclease)
Pharmacokinetics
- rapidly cleared after administration[1]
Adverse effects
- transient infusion-related reactions[1] Mechanism: of action:
- CRISPR-Cas9 with two components:
- single-guide RNA that precisely targets TTR
- human-codon-optimized mRNA sequence of Streptococcus pyogenes Cas9 protein
- lipid-nanoparticle formulation developed for efficient delivery to hepatocytes[1] the main source of circulating TTR protein.
- a single dose of nex-z is associated with rapid, & durable reductions in serum TTR
More general terms
References
- ↑ 1.0 1.1 1.2 1.3 Fontana M, Solomon SD, Kachadourian J et al CRISPR-Cas9 Gene Editing with Nexiguran Ziclumeran for ATTR Cardiomyopathy. N Engl J Med. 2024 Nov 16. PMID: https://www.ncbi.nlm.nih.gov/pubmed/39555828 https://www.nejm.org/doi/10.1056/NEJMoa2412309