The PaVe-GT team has gained widely applicable first-hand experience in gene therapy development and regulatory interactions and is committed to publicly sharing scientific and regulatory lessons learned in developing four rare disease gene therapies. This is a unique feature and an integral component of the program.
This page provides translatable resources to promote the development of other gene therapies. Use these PaVe-GT regulatory packages, target product profiles, templates and more, as examples to inform your gene therapy program.
This section includes a template and TPP examples used to guide the PaVe-GT program.
This section includes an INTERACT package template, example regulatory documents submitted to the FDA from the PaVe-GT PCCA program and FDA’s feedback.
Template for INTERACT Meeting Request // FDA INTERACT Meeting Communications for AAV9-hPCCA
This section includes examples of successful applications submitted to the FDA for an Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD), as well as templates for these applications.
This section features infographics that illustrate the various activities in the translation from discovery to clinical testing for gene therapies.
Translational Research Process
Proof of Concept
Manufacturing
Toxicology
Bioanalytical Methods
Clinical Testing
Regulatory Strategy
These resources will be shared over the course of the PaVe-GT program: