Bernard Gilly, Gensight CEO (Eric Piermont/AFP via Getty Images)

They thought their gene ther­a­py failed. In­stead, it spawned a med­ical mys­tery

José-Alain Sa­hel was on a rare va­ca­tion in Por­tu­gal in the spring of 2018 when his phone rang with grim news: The gene ther­a­py he had worked on for a decade, a po­ten­tial cure for a rare form of blind­ness, had failed in a piv­otal tri­al.

“In the first minute, I was very dis­ap­point­ed,” Sa­hel says. “I said, well OK, it’s not work­ing.”

A failed tri­al in drug de­vel­op­ment is crush­ing but not un­ex­pect­ed, a trade­off of do­ing busi­ness in bi­ol­o­gy. You ex­am­ine the full da­ta, go back to the draw­ing board and ei­ther aban­don the ef­fort or tweak and try again. Sa­hel, founder of four com­pa­nies and the long­time head of the Vi­sion In­sti­tute of Paris, was used to the process. But this time, when the full da­ta came, he was be­wil­dered.

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