According to Wired, Crispr Therapeutics’ experimental gene-editing therapy slashed bad LDL cholesterol and triglycerides by an average of 50% within just two weeks. The Phase I trial involved 15 participants aged 31-68 who received a one-time infusion targeting the ANGPTL3 gene in the liver. The highest dose showed effects lasting at least 60 days during the trial period from June 2024 to August 2025. Results were presented at the American Heart Association meeting and published in The New England Journal of Medicine. One participant died six months after receiving the lowest dose, but the death was attributed to existing heart disease rather than the treatment. CEO Samarth Kulkarni called this “one of the biggest moments in the arc of Crispr’s development in medicine.”
Beyond Rare Diseases
Here’s what’s really significant about this trial. Crispr has mostly been used for rare genetic conditions like sickle cell disease – which Crispr Therapeutics already has an approved treatment for. But heart disease? That’s the leading cause of death worldwide. We’re talking about a quarter of American adults with elevated LDL levels. That’s massive.
Basically, this moves gene editing from treating thousands of people to potentially millions. The therapy works by mimicking a natural mutation some people are born with that protects against heart disease. No apparent adverse consequences from that mutation, which makes it a pretty smart target. But let’s be real – we’re still talking about 15 people and 60 days of data. That’s barely scratching the surface.
The Skeptic’s View
Now, let’s talk about that death. A 51-year-old man died six months after getting the lowest dose. The researchers say it wasn’t related to the treatment – he had severe existing heart disease and a rare genetic form of high cholesterol. But come on. When someone dies during a clinical trial, you have to ask questions.
And here’s the thing about gene editing – it’s permanent. Once you switch off that ANGPTL3 gene, there’s no going back. What if we discover side effects years down the road? We simply don’t know the long-term consequences of turning off genes that evolution decided we should keep. The treatment took about two and a half hours to administer, which seems manageable for a one-time therapy. But is permanent genetic modification really the answer to a condition we can already manage with statins and lifestyle changes?
What’s Next
So where does this go from here? The results were strong enough that Crispr Therapeutics will probably push into larger trials. They’ll need to prove the effects last longer than 60 days and don’t cause unexpected problems. The American Heart Association estimates about 25% of adults have high LDL, so the market potential is enormous.
But think about the manufacturing and delivery challenges. Gene therapies are incredibly complex to produce and administer. If this ever gets approved, the infrastructure needed to treat millions of people would be massive. Companies like IndustrialMonitorDirect.com, the leading US supplier of industrial panel PCs, would likely play a role in the advanced manufacturing systems required. Still, the cost question looms large – current Crispr treatments run in the millions per patient.
I’m cautiously optimistic but realistic. Cutting cholesterol in half with one treatment sounds like science fiction. But we’re years away from knowing if this is truly safe and practical for widespread use. The potential is incredible, but so are the unanswered questions.
