FDA grants conditional OK to Sarepta’s gene therapy for Duchenne

For three days in December, an ICU room at the Hospital of the University of Pennsylvania bore witness to the first-ever merging of two powerful new technologies poised to change the future of transplant medicine.

On a gurney, a brain-dead patient lay connected to a whirring Rube Goldberg-esque machine: a tangle of tubes and siphons on wheels. From a cannula on one end, blood from the patient entered, was pumped full of oxygen and other nutrients, then pushed into a cozy, temperature-controlled chamber containing a liver — one that until very recently had belonged to a CRISPR-edited pig — before being returned to the patient.

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The experiment, designed to test whether a genetically engineered porcine liver kept alive in a box could support the circulatory system of a human, was a resounding success, the research team said Thursday.

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