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Gene Therapy: AAV Doses Should Not Be Subject To Fixed Upper Limit, US FDA Panel Says

Executive Summary

Lack of reference standards inhibits ability to make comparisons between products or set caps for total vector genome dose or total capsid dose, advisory committee says; panelists suggest other approaches to prevent and mitigate toxicities, including running longer-term animal studies and investigating the efficacy of immunosuppression prophylaxis and treatment strategies.

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