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This article shares the PaVe-GT team’s experience of pre-IND and Type C meetings with the FDA for its propionic acidemia gene therapy candidate. The article discusses lessons learned from the development of the first AAV9 gene therapy in the platform, FDA feedback on pharmacology/toxicology studies, manufacturing/product quality and first-in-human study design, use of biomarkers, and leveraging clinical trial evidence for future development. The article also shares the related regulatory documents and templates.
Human Gene Therapy | June 19, 2026
