Health Affairs January 5, 2024
The emergence of cell and gene therapies (CGTs) has been a source of hope for patients living with once-uncurable diseases. Gene therapy involves the introduction of genetic material, usually in a carrier or vector, to the appropriate cells of the body; the goal is to treat disease by, for example, increasing the production of healthy proteins or decreasing the production of disease-causing proteins. Cell therapy aims to treat a disease through modification of a patient’s own cells or transfer of donor cells, such as immune cells, into a patient.
Unfortunately, these blockbuster therapies often make headlines not because of their innovative technology but because of their high price. For example, Zolgensma, a gene therapy for treating spinal muscular atrophy priced...
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