Christopher D: Personalized CRISPR Therapy and the Future of Rare Disease Care
Christopher D., Executive Specialty Sales at AbbVie, shared a post on LinkedIn:
“A baby named KJ Muldoon is why I can’t stop thinking about where medicine is headed.
KJ was born with a rare genetic disorder that stopped his liver from clearing ammonia from his blood.
The old options: a transplant, or irreversible damage. Instead, his team built a personalized CRISPR therapy for his exact mutation and delivered it straight to his liver.
It worked he’s the world’s first patient treated this way.
That case is part of a bigger shift in liver and rare disease care right now:
Base or prime editing moving from lab to bedside for hemophilia, metabolic disorders
AI catching fatty liver disease earlier and personalizing treatment
Liver organoids letting researchers test therapies before they reach a patient
The liver’s biology making it a prime target for next gen gene therapy
‘Rare disease’ used to mean ‘no good options.’ That’s changing fast.
What’s the most exciting thing you’ve seen in this space lately?
Sources:
NIH, NCATS, Baby KJ story:
Jackson Laboratory, gene editing for rare liver disorder”
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