Gene therapy for inherited blindness sets precedent: $850,000 price tag

A landmark gene therapy to treat a rare, inherited form of blindness will cost $850,000 — a price tag so daunting that its maker will offer health insurers partial rebates if the drug doesn't work and is seeking to pilot an installment payment option.

The drug, called Luxturna, is the realization of a long-sought scientific dream: The one-time treatment corrects a faulty gene to improve vision, allowing patients to see the stars or their parents' faces. Only 1,000 to 2,000 people in the United States are thought to have deteriorating vision caused by this errant gene, called RPE65, but Luxturna is widely expected to be the first in a wave of cutting-edge treatments that are targeted at fixing the causes of a wide range of genetic diseases — while also raising difficult questions about how to pay for them.

The idea that drugs can be made to correct the defective genes that lie at the root of inherited diseases has long tantalized medicine. Nearly three decades ago, gene therapy was first tested in a patient in the U.S., but after a teenager named Jesse Gelsinger died in a clinical trial in 1999, the field screeched to a...