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Real and Pricey

Gene therapy is becoming more of a reality, writes Michael White at Pacific Standard.

He notes that 2017 brought the approval by the US Food and Drug Administration of three gene therapies: Novartis' Kymriah for acute lymphoblastic leukemia, Kite Pharmaceutical's Yescarta for large B-cell lymphoma, and Spark Therapeutics' Luxturna for retinal dystrophy.

White notes that gene therapy has long been sought after — and had come to represent "unfulfilled promises of biomedical scientists" — as researchers have been noting disease genes for a hundred years, but hopes about gene therapy were dashed in the 1990s when a patient suffered a fatal reaction. But now, White says researchers have solved some of the problems of gene therapy.

But, there are remaining issues, he notes, including how to pay for it. The newly approved treatments are being priced in the range of hundreds of thousands of dollars.

"Compared to the cost of a lifetime of drugs or disability accommodations, perhaps nearly $1 million for a cure isn't unreasonable," White writes. "On the other hand, as more gene therapies reach the clinic, such astronomical prices could raise insurance premiums and put these cures out of reach for some."