U.S. Department of Health and Human Services

National Institutes of Health

National Center for Advancing Translational Sciences

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Publications

This page lists project-relevant, peer-reviewed scientific articles from the PaVe-GT team along with the research impact (Altmetric data).

Adeno-Associated Virus Gene Therapy Translation: Lessons from Early Regulatory Meetings

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

Adeno-Associated Virus Gene Therapy Development: Early Planning and Regulatory Considerations to Advance the Platform Vector Gene Therapy Program

This article shares the team’s insights on early planning and navigating regulatory requirements for gene therapy development. It highlights the value of a target product profile and INTERACT meeting with the FDA.

Human Gene Therapy | March 6, 2025

Successfully Navigating FDA Orphan Drug and Rare Pediatric Disease Designations for AAV9-hPCCA Gene Therapy: The NIH PaVe-GT Experience

This article provides an overview of the application process for FDA incentive programs that facilitate orphan drug development. Orphan Drug and Rare Pediatric Disease Designation requirements, examples, and relevant resources are shared.

Human Gene Therapy | March 20, 2023

The Platform Vector Gene Therapies (PaVe-GT) Project: Increasing the Efficiency of Adeno-Associated Virus (AAV) Gene Therapy Clinical Trial Startup

This article describes the challenges in developing gene therapies and the goal of the PaVe-GT program.

Human Gene Therapy | October 16, 2020