Andreessen Horowitz December 8, 2023
Originally discovered in bacteria, CRISPR quickly became the workhorse gene-editing system underlying the next generation of programmable medicines. Today’s landmark approval by the US Food and Drug Administration of Vertex Pharmaceuticals’ and CRISPR Therapeutics’ Casgevy, the world’s first CRISPR-based treatment, marks a groundbreaking shift in how we tackle intractable diseases.
And it’s a milestone barely a decade in the making: the original publications describing CRISPR’s potential date back to 2012. Since then, CRISPR has transitioned from being a bacterial protein to a gene-editing tool, earned a Nobel Prize, and is now an FDA-approved medicine.
On the “cutting” edge
CRISPR uses a protein called Cas9 to cut DNA at precise locations to treat diseases. Casgevy treats sickle cell disease by targeting...
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