FDA Includes UF Researcher As Advisory Board Considers Gene Therapy Risks

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 Sept. 2-3, 2021. 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.

Coming in the shadow of preclinical red flags, safety alerts, clinical holds, and patient deaths, the meeting is designed to go through any and all toxicity risks related to AAV. And the FDA wants to get input on how it should deal with each of those issues, with regulatory implications on everything from mouse studies to clinical trials to manufacturing standards.

Advisory committee member Kenneth Berns of the University of Florida, one of the original researchers in AAV, has declared a conflict of interest due to grants or stock holdings in affected companies. He, along with Roland Herzog at Indiana University and Charles Vite at the University of Pennsylvania, were granted waivers to participate in the panel.

Read more about Capping AAV Dose? Tweaking Gene Therapy Trials? New Animal Models? FDA Poses Far-Reaching Questions for Experts.