Gen­Sight's gene ther­a­py mys­tery con­tin­ues, as pa­tients im­prove in an­oth­er failed tri­al

A three-year-old med­ical mys­tery took its lat­est turn Thurs­day, when Gen­Sight re­leased da­ta from its third tri­al for a rare form of blind­ness: Once again pa­tients saw their vi­sion im­prove in both eyes. And once again the tri­al tech­ni­cal­ly failed.

The Phase III study was for Lumevoq, a gene ther­a­py de­signed to fix Leber hered­i­tary op­tic neu­ropa­thy, or LHON, a rare ge­net­ic dis­ease that caus­es rapid vi­sion loss in pa­tients dur­ing adult­hood. It had been Gen­Sight’s lead pro­gram, lift­ing the com­pa­ny to a €40 mil­lion IPO, but when they com­plet­ed the piv­otal tri­al in 2018, they found para­dox­i­cal re­sults.

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