Today’s newsletter brings you two stories — one about a stunning use of CRISPR to save a baby’s life, and the other about a cholesterol drug that might finally redeem a long-abandoned class.
Both are reminders that medical breakthroughs are rarely straightforward — and often slower than the headlines suggest.
🧬 Personalized Gene Editing Just Saved a Life — But It’s Not a Blueprint (Yet)
A baby with a fatal metabolic condition may have been cured using a personalized CRISPR therapy — and that’s not hyperbole.
You may have seen this story covered in New York Times and the full case report was just published the New England Journal of Medicine.
The condition: a deficiency in carbamoyl-phosphate synthetase 1, a rare enzyme defect that disrupts protein metabolism and typically leads to early death or permanent disability.
The baby received a one-time, gene-edited therapy targeting the liver — and has survived nearly a year with dramatically improved metabolic function.
It’s an incredible result. And it’s a major proof of concept that gene editing can be personalized and deployed to treat an otherwise untreatable disease.
This is incredibly exciting and it’s also inspiring - there’s promise that technology like this could potentially help the millions of people born with rare defects of metabolism.
But before we start projecting this into a CRISPR-fueled future, some perspective is important.
Why this case worked so well
There are a few factors that made this almost a best case scenario for gene editing:
The liver is an accessible target for genetic therapies.
Only a small amount of enzyme activity was needed to make a clinical difference.
The disease was universally fatal without treatment, making the risk/benefit equation very different from most conditions.
When there are no other treatment options, and the disease is lethal, the bar for trying something experimental is much lower.
That point - the risk/benefit analysis really matters for something like this - is vital when considering how likely CRISPR technology is to be widespread anytime soon to intervene on human disease.
That’s part of why this case succeeded — not just because of the gene-editing technology, but because it was deployed under ideal biological and ethical conditions.
What we still don’t know
Off-target effects of gene editing are not fully understood.
Durability of the intervention is unknown — will the effect persist for years?
Long-term consequences (cancer risk, immune response, infection susceptibility) are still theoretical — but real.
And also keep in mind that almost every other medical condition is more complicated from a genetic perspective.
The deeper you dive into genetics, the more complicated the stuff gets.
Most common conditions — from heart disease to diabetes to neurodegenerative disorders — are polygenic, with dozens or hundreds of contributing genes, epigenetic interactions, environmental influences, and unknown trade-offs.
The idea that we’ll “edit” our way out of these conditions anytime soon is total science fiction.
Gene editing will be transformative — but only for rare, well-understood, single-gene diseases.
For now, that includes conditions like sickle cell anemia, some inborn errors of metabolism, and potentially a few immune deficiencies. Not much else.
So yes — this is the future.
But like most of medicine, it’s just not evenly distributed yet.
❤️ Obicetrapib: A Cholesterol Drug That Deserves a Second Look
Switching gears — let’s talk about a drug I’m really excited about.
Obicetrapib is a new CETP inhibitor — a class of drugs previously written off after high-profile failures. Drugs like torcetrapib were shown to raise HDL and lower LDL but didn’t reduce cardiovascular events — and in some cases, increased them due to off-target effects.
But obicetrapib might be different.
The phase 3 BROADWAY trial showed that obicetrapib significantly lowers LDL — even in patients already on statins and ezetimibe.
We’re still waiting on the most important data — the PREVAIL trial, which will tell us whether those LDL reductions actually reduce heart attacks, strokes, and cardiovascular death. That trial is expected to read out in the next 1–2 years.
If it’s positive, this would be a big win:
A potent oral LDL-lowering therapy
With a distinct mechanism of action
That could serve patients who need add-on therapy or have statin intolerance
We’re essentially just one step away from knowing whether this drug works to reduce heart attacks, strokes, and death.1
I’ll be watching PREVAIL closely.
🧠 Final Thought
Both stories — the CRISPR baby and the cholesterol drug — offer a lesson in how progress happens in medicine.
Not in sweeping revolutions. But in small, specific advances, often in narrow patient populations, that inch us forward.
And occasionally, one of them surprises us.
Death is hard to show a benefit in, because you need a really big trial that follows patients for a sufficient amount of time.