The Western world’s first gene therapy is soon to become but a footnote in biotech history, doomed by minuscule demand and a colossal price. But its failure has hardly dampened the enthusiasm of the scientists and biotech companies betting that gene therapy’s best days are ahead.
The therapy, made by the Dutch biotech UniQure, treats a one-in-a-million rare disease called familial lipoprotein lipase deficiency and has been used exactly once since winning European approval in 2012, the company said. That’s in part because it costs about $1 million for a one-time dose, a price that made it nearly impossible to get insurance coverage, as MIT Technology Review reported last year.
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