PaVe-GT Resources

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.

Target Product Profile (TPP)

This section includes a template and TPP examples used to guide the PaVe-GT program.

Template for TPP // TPPs for PaVe-GT Candidates

INTERACT Meeting with FDA

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

FDA Incentive Programs for Rare Disease Drug Development

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.

Template for ODD // Template for RPDD

ODD Request for AAV9-hPCCA // RPDD Request for AAV9-hPCCA

Upcoming Resources

These resources will be shared over the course of the PaVe-GT program:

Preclinical: study designs, vector designs, animal models and proof of concepts, manufacturing processes and critical quality attributes, bioanalytical methods, and toxicology study results, including biodistribution
Regulatory: regulatory strategies, templates and examples of regulatory packages, and related FDA feedback
Clinical: clinical protocol designs and templates, surrogate endpoint approaches, and clinical research operations for gene therapy studies