Gene ther­a­py from Bio­gen's $800M buy­out flops in mid-stage study, deal­ing blow to new am­bi­tions

The #2 can­di­date from Bio­gen’s $800 mil­lion oc­u­lar gene ther­a­py buy­out has failed in a mid-stage tri­al, deal­ing an ear­ly blow to the big biotech’s plans to re­vi­tal­ize its pipeline with new tech­nolo­gies.

Bio­gen an­nounced that the can­di­date, an ex­per­i­men­tal treat­ment for a rare and pro­gres­sive form of blind­ness called X-linked re­tini­tis pig­men­tosa (XL­RP), failed to suf­fi­cient­ly im­prove vi­sion in pa­tients’ treat­ed eye — pa­tients on­ly re­ceived an in­jec­tion in one eye — af­ter a year, on a stan­dard scale, com­pared to their un­treat­ed eye. The com­pa­ny said they saw “pos­i­tive trends” on sev­er­al sec­ondary end­points, in­clud­ing vi­su­al acu­ity, but de­clined to say whether the tri­al ac­tu­al­ly hit any of those end­points.

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