A gene ther­a­py mys­tery? Gen­Sight blast­ed as 1st PhI­II eye study ends in an in­trigu­ing fail­ure

Gen­Sight has a gene ther­a­py mys­tery on its hands, which is about the last thing it want­ed at the end of Phase III.

As­sess­ing the 48-week da­ta from its first piv­otal test of a treat­ment for Leber Hered­i­tary Op­tic Neu­ropa­thy — or LHON, a rare ge­net­ic dis­ease that caus­es blind­ness — in­ves­ti­ga­tors pulled out a mean­ing­ful im­prove­ment in sight in their one treat­ed eye.  

The mys­tery: The oth­er eyes that re­ceived a sham ther­a­py did just as well with their eye­sight. Ex­act­ly as well.

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