Verve Therapeutics dosed the first patient with a first-in-class base editing heart drug — a treatment the genetic medicines company hopes will be "game-changing" to the traditional chronic care model.
The dosing officially began Verve’s heart-1 trial, which will examine VERVE-101 for the treatment of a genetic subtype of atherosclerotic cardiovascular disease, heterozygous familial hypercholesterolemia (HeFH).
The novel investigational gene editing medicine is designed to be a single-course treatment that can permanently silence the PCSK9 gene in the liver. Changing the expression of this gene, according to Verve, brings down disease-causing cholesterol levels.
Verve says the drug has the potential to offer HeFH patients a single-course, life-long treatment solution. VERVE-101 is a base editing medicine, intended to make a single spelling change to prevent the disease-causing expression.
When successful, gene and cell therapy drugs bring hope about a future where certain diseases could potentially be eradicated. But fulfilling this promise requires many hands and brains that the industry is having a hard time finding. Read more about this in our recent cover story.