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A custom-made drug. Developed for one baby. Delivered in just six months.
 
A custom-made drug.
 
Developed for one baby.
Delivered in just six months.
 
This isn’t a pitch deck. It’s what actually happened.
 
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A newborn was diagnosed with CPS1 deficiency — a rare genetic condition that causes toxic ammonia to build up in the body.
There was no approved treatment. No case study. No roadmap.
 
Doctors had two choices:
1.Wait for science to catch up
2.Push science forward
 
They chose option two.
 
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Using CRISPR base editing, scientists corrected the baby’s unique mutation — not by cutting the DNA, but by swapping out a single faulty letter in his genetic code.
 
One problem: this mutation had never been treated before.
So they created a brand-new guide RNA molecule, tailored just for him.
They called it “kayjayguran” — the only one of its kind on Earth.
 
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Here’s where it gets even more groundbreaking:
 
Most gene therapies take years to develop.
This one was ready in six months.
 
They used mRNA technology and lipid nanoparticles — similar to what powered COVID-19 vaccines — to deliver the treatment directly to the baby’s liver.
 
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What happened next?
•Ammonia levels dropped
•He started tolerating food
•No major side effects
 
A single shot. A textbook response. A life saved.
 
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This is called N-of-1 medicine — therapy designed for one patient, one mutation, one solution.
 
For decades, we’ve talked about personalized medicine.
This baby’s story proves it’s not a theory anymore. It’s happening.
 
⸻
 
Imagine a world where every rare disease has a custom treatment.
Not in 20 years.
Now.
 
This is not the future of medicine.
It’s the present — and it’s only just beginning.
 
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If you’re in biotech, healthcare, or science communication — this is the kind of story we need to be paying attention to.
 
Because the next N-of-1 patient could be anywhere.
And science just showed us that one patient is enough to move the needle for the world.
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