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Gene therapy may soon be more affordable

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Gene therapy, which introduces new genetic material into a person’s DNA, was developed as a revolutionary way to treat some of the rarest syndromes on earth. Yescarta is the second example of a gene therapy that modifies the DNA in a person’s immune cells to go after cancer.

To see if there’s a connection between the price of a gene therapy and the estimated number of patients it will treat per year, we decided to plot those figures for the approved gene therapies in the U.S. and Europe and one that’s pending approval in the U.S. Herman Sanchez, managing partner at life science consulting firm Trinity Partners, says there is a correlation between the patient population and price of the therapy: the fewer eligible patients, the more expensive it is.

In the U.S., Novartis says about 300 patients a year will be eligible for its gene therapy Kymriah, which treats a type of childhood leukemia.

Though Yescarta’s price still prompted outcries from patient advocates, it bodes well for gene therapies in the pipeline for diseases with more patients, like hemophilia and sickle-cell, which affect about 20,000 people and 100,000 in the U.S., respectively.

Next up for approval is a gene therapy called Luxturna that restores some vision in patients with an inherited type of blindness.

Spark says about 1,000 to 2,000 total people in the U.S. could be eligible for its therapy, which improves sight by replacing a mutated gene in the retinal cells.


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