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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