Four Children Die from Gene Therapy Known as AT132—Will the FDA Move In & Halt?

Recently TrialSite reported on the U.S. Food and Drug Administration (FDA) gene therapy meeting titled "Cellular, Tissue, and Gene Therapies Advisory Committee" September 2-3, 2021, centering on emerging regulatory issues, particularly safety, associated with the rapidly growing field of gene-based therapy development. This revolutionary concept has already led to some approved therapies. The focus of this recent session included the safety of adeno-associated viruses (AAVs), a common gene therapy approach. Of noted concern were recent clinical holds such as with BioMarin’s gene therapy for phenylketonuria, a rare disease and one associated with bluebird’s clinical study involving elivaldogene autostemcel (eli-cel, Lenti-D)—an investigational gene therapy for cerebral adrenoleukodystrophy (CALD). TrialSite concluded that while the FDA wasn’t stopping gene therapy programs due to high risk, they suggested the industry proceed with great caution. In parallel, however, one investigational gene therapy is associated with four young children’s death. During the second half of 2020, Astellas Pharma put a hold on screening and dosing more participants in a gene therapy-based study after ...

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