Cap­ping AAV dose? Tweak­ing gene ther­a­py tri­als? New an­i­mal mod­els? FDA pos­es far-reach­ing ques­tions for ex­perts

Is it time to set a lim­it for how high a dose gene ther­a­py de­vel­op­ers and in­ves­ti­ga­tors are al­lowed to give pa­tients in each tri­al?

That’s one of the ques­tions the FDA is pos­ing to its Cel­lu­lar, Tis­sue, and Gene Ther­a­pies Ad­vi­so­ry Com­mit­tee as ex­perts pre­pare for a two-day meet­ing next week. De­pend­ing on how the dis­cus­sion goes — and what the agency makes of it — the re­sults could com­plete­ly re­de­fine the rules for a ma­jor sec­tion of the boom­ing gene ther­a­py field: treat­ments that are de­liv­ered by ade­no-as­so­ci­at­ed virus vec­tors.

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