2 weeks ago
Single-Shot Gene Editing Treatment Targets Cholesterol Through PCSK9
LDL is often called bad cholesterol.
People with very high LDL commonly take statin medicines for many years.
Researchers are testing a different idea called gene editing.
Verve-102 is an experimental treatment for this purpose.
It would be given through one intravenous infusion.
The treatment is designed to work on the PCSK9 gene in liver cells.
It uses a method called in vivo base editing.
This method aims to make a precise change to DNA inside the body.
The goal is to lower LDL cholesterol after a single treatment.
Verve-102 is an experimental treatment designed to lower LDL cholesterol after one intravenous infusion.
The treatment targets the PCSK9 gene in liver cells.
It uses in vivo base editing to make a precise DNA change inside the body.
People with very high LDL cholesterol commonly take statins regularly for years.
Researchers are exploring gene editing as an alternative approach to long-term cholesterol medication.
- Who
- Researchers exploring gene-editing treatment for people with very high LDL cholesterol.
- What
- Verve-102 is an experimental gene-editing treatment designed to lower LDL cholesterol after one intravenous infusion.
- Where
- Inside liver cells, using an intravenous infusion to deliver the treatment into the body.
- When
- Why
- To explore a potential way to lower LDL cholesterol without requiring regular statin treatment for years.
Key facts
- Treatment
- Verve-102
- Target
- The PCSK9 gene in liver cells
- Editing method
- In vivo base editing
- Administration
- A single intravenous infusion
- Intended effect
- Lower LDL cholesterol
- Current status
- Experimental






