DB18525InvestigationalGene Therapies
NTLA-2001 is a clustered regularly interspaced short palindromic repeats CRISPR/ Cas9-based gene therapy consisting of a single guide RNA targeting the human TTR gene and a messenger RNA encoding Cas9.
- Also known as
- nexiguran ziclumeran
- Code names
- NTLA-2001
What you can answer from here — as of April 23, 2025
Which companies are running trials of NTLA-2001, in which indications and phases, and which of those programs are still active?
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