FDA chief Got­tlieb is build­ing a reg­u­la­to­ry speed­way to ac­cel­er­ate gene ther­a­py de­vel­op­ment

In a ral­ly­ing cry for gene ther­a­py, FDA Com­mis­sion­er Scott Got­tlieb says he’s de­ter­mined to clear the path­way for drug de­vel­op­ers in a move to ac­cel­er­ate the first wave of gene ther­a­pies point­ed to the mar­ket.

The first ther­a­peu­tic area to ben­e­fit from new sur­ro­gate end­points will be he­mo­phil­ia, Got­tlieb said — im­me­di­ate­ly ring­ing a bell for com­pa­nies like Spark Ther­a­peu­tics $ONCE, Pfiz­er $PFE, Bio­Marin $BM­RN and uniQure $QURE, which are de­vel­op­ing cures for both ver­sions of the bleed­ing dis­or­der. Un­der the yet-to-be-an­nounced guide­lines, fac­tor pro­duc­tion may in some cas­es be suf­fi­cient as a mea­sure of ben­e­fit.

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