One shot genetic cures? (

$3.5 million for a one-time dose? Are you kidding? But then … maybe it is reasonable?

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Hemgenix now produces a genetic cure. For those with hemophilia B, a disease that causes spontaneous bleeding due to a DNA mutation, there is now a treatment that injects a normal gene into the patient’s body to start manufacturing a corrected protein, with a 94% rate of no longer needing Factor IX infusions. Read it. Amazing.

And yes $3.4 million is a huge cost for a one-time treatment, but that is comparable and cheaper, than a lifetime of constant infusions and bleeding episodes and complications. And the cost WILL come down. Science fiction, here we come.

Author: CT Lin

CMIO, UCHealth (Colorado); Professor, University of Colorado School of Medicine

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