DB17597InvestigationalGene Therapies
NTLA-2002 is a lipid nanoparticle encapsulating single guide RNA (G012267) targeting the human KLKB1 gene and messenger RNA (mRNA000042) encoding Cas9. It is an investigational therapy designed to knock out... It is an investigational therapy designed to knock out the target gene kallikrein B1 (KLKB1) to reduce plasma kallikrein activity, thereby preventing hereditary angioedema attacks.
- Also known as
- lipid nanoparticle encapsulating single guide RNA (G012267) targeting the human KLKB1 gene and messenger RNA (mRNA000042) encoding Cas9
- Code names
- NTLA-2002
Resolves to
Compound
What you can answer from here — as of April 23, 2025
Which companies are running trials of NTLA-2002, in which indications and phases, and which of those programs are still active?
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