CTX320 is an in vivo liver gene therapy that directly edits the LPA gene in patients, aiming to reduce lipoprotein(a) (Lp(a)), a genetically determined risk factor for cardiovascular disease.
Phase 1 ongoing; 73% Lp(a) reduction; H1 2026 update planned
Checked 10 Oct0 updates this week
CTX320 uses lipid nanoparticles to deliver CRISPR machinery to liver cells, where it edits the LPA gene.
The LPA gene encodes lipoprotein(a), a cardiovascular risk factor that lacks approved treatments.
One-time editing results in durable reduction of Lp(a) levels, potentially lasting years after a single dose.
| Spec | CTX320 |
|---|---|
| Mechanism | In vivo CRISPR/Cas9 gene editing of LPAR (reported) |
| Lp(a) reduction (dose escalation) | Up to 73%R (reported) |
| Target population | ~20% of global populationR (reported) |
R reported by the company
CRISPR Therapeutics
CRISPR, Casgevy, in vivo liver editing
Swiss-American gene-editing company co-founded by Nobel winner Emmanuelle Charpentier. With Vertex it made Casgevy, the first approved CRISPR medicine, for sickle cell disease and beta thalassemia. Now it edits liver genes to treat heart disease.
