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

 
• By Sue Sutter

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.

Dime
Manufacturers cannot just "turn on a dime" and make a new lot of AAV gene therapy with much greater purity, the FDA acknowledges. • Source: Alamy

The US Food and Drug Administration appears unlikely to impose a blanket cap on either the total vector genome dose per subject or the total capsid dose for adeno-associated virus (AAV) vector-based gene therapies in an attempt to limit toxicity, based upon input received from the agency’s external experts.

However, members of the Cellular, Tissue and Gene Therapies Advisory Committee called for more standardization and better approaches to product characterization in the AAV gene therapy field to better ascertain what role, if any,

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