VERVE-101 and VERVE-102 are an experimental

gene therapy Gene therapy is a medical field which focuses on the genetic modification of cells to produce a therapeutic effect or the treatment of disease by repairing or reconstructing defective genetic material. The first attempt at modifying human DN ...

developed by Verve Therapeutics that targets the

PCSK9 Proprotein convertase subtilisin/kexin type 9 (PCSK9) is an enzyme encoded by the ''PCSK9'' gene in humans on chromosome 1. It is the 9th member of the proprotein convertase family of proteins that activate other proteins. Similar genes (ortholog ...

gene and is intended to reduce blood cholesterol levels. It works on the same protein as the cholesterol-lowering drugs known as

PCSK9 inhibitors Proprotein convertase subtilisin/kexin type 9 (PCSK9) is an enzyme encoded by the ''PCSK9'' gene in humans on chromosome 1. It is the 9th member of the proprotein convertase family of proteins that activate other proteins. Similar genes (ortholog ...

but, unlike them, is permanent. It works via base editing, a form of

CRISPR gene editing CRISPR gene editing (pronounced "crisper") is a genetic engineering technique in molecular biology by which the genomes of living organisms may be modified. It is based on a simplified version of the bacterial CRISPR-Cas9 antiviral defense sys ...

. It is one of the first gene therapies that could be beneficial to a wider segment of the population, in contrast to earlier gene therapies that were developed to treat a rare genetic disorder. Both treatments use the same RNA gene editing technology, but they use a different

lipid nanoparticle lipid nanoparticles (LNPs), are nanoparticles composed of lipids. They are a novel pharmaceutical drug delivery system (and part of nanoparticle drug delivery), and a novel pharmaceutical formulation. LNPs as a drug delivery vehicle were f ...

delivery vehicle.

References

{{reflist PCSK9 inhibitors Experimental gene therapies