Bio­Marin gene ther­a­py could be on hold 'for sev­er­al quar­ter­s' af­ter FDA man­dates new pre­clin­i­cal tests

The FDA is stay­ing cau­tious af­ter da­ta came out sug­gest­ing that one of Bio­Marin’s ex­per­i­men­tal gene ther­a­pies might have the po­ten­tial to dri­ve can­cers.

Al­though de­tails re­main sparse, Bio­Marin said on Fri­day that its gene ther­a­py for the rare dis­ease phenylke­tonuria, or PKU, will re­main on hold af­ter the FDA asked for ad­di­tion­al non-clin­i­cal — i.e., lab-based or an­i­mal stud­ies — to as­sess “the the­o­ret­i­cal onco­genic risk to hu­man study par­tic­i­pants.”

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