“CTX310 works by switching off a gene called ANGPTL3 through a single, two-hour infusion. It was inspired by studies showing people born with an inactive version of the ANGPTL3 gene have a lower lifetime risk of heart disease with no apparent adverse consequences.

Regeneron’s Evkeeza, which treats a rare genetic disorder called homozygous familial hypercholesterolemia, targets the same gene but requires monthly infusions.

CRISPR’s trial of 15 patients aged 31-68, conducted in Australia, New Zealand and the UK, tested five different doses. All participants had high triglycerides, high LDL cholesterol or both and had failed to respond to other treatments.

Among four patients who received the highest dose, triglycerides on average were cut by 55% and LDL by 50% two weeks after treatment. Levels stayed low for at least two months.”

From Reuters.