CRISPR therapy keeps cholesterol down a year after one dose

CRISPR Therapeutics gave patients a single dose of an experimental gene-editing drug over a year ago. Their cholesterol and triglycerides are still down, the company said Friday. The drug, called CTX310, uses the gene-editing tool CRISPR-Cas9 to permanently switch off part of a gene called ANGPTL3. That gene normally raises levels of fat-related molecules in the blood tied to heart disease. One shot, and the effect held for a full year.

High cholesterol and triglycerides raise the risk of heart attacks and strokes for millions of people. Current treatments, like statins or PCSK9 inhibitor shots, require taking a pill or a shot regularly. Often, that means doing it for life. CTX310 is designed to work with a single treatment instead. That's the appeal of gene editing broadly. Fix the gene once, and the problem stays fixed. But this trial only tested a small number of patients so far. And one year of data isn't the same as ten. Serious side effects could still show up later. And CRISPR edits can't be undone if something goes wrong.

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Will this hold up in a larger trial? CRISPR Therapeutics still needs to test CTX310 in far more patients. Only then would regulators even consider approving it. The company is also racing rivals working on similar one-time cholesterol treatments. Timing matters here. If later trials confirm this, it could replace a lifetime of pills for people at high risk of heart disease. That's still years away, and depends on results nobody has seen yet.