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NIH Research Festival

September 23 – 25, 2024

AAV gene therapy development: Early planning and regulatory considerations for advancing the NIH Platform Vector-Gene Therapy (PaVe-GT) Program

Authors

  • RM Lomash
  • J Dehdashti
  • OA Shchelochkov
  • RJ Chandler
  • L Li
  • I Manoli
  • J Sloan
  • P Terse
  • X Xu
  • R Stan
  • D Saade
  • PJ Brooks
  • DC Lo
  • CG Bonnemann
  • CP Venditti
  • AR Pariser
  • EA Ottinger

Abstract

Gene therapy development presents multiple challenges and early planning is vital in the successful implementation of such programs. The Platform Vector-Gene Therapy (PaVe-GT) program is a NIH initiative developing adeno-associated virus (AAV) gene therapies for four rare diseases. Utilizing the platform-based approach, the program aims to incorporate efficiencies throughout the preclinical and clinical development processes followed by public dissemination of scientific and regulatory learnings. Early in development, the establishment of a Target Product Profile (TPP) by the research team is a critical step to guide product development and align preclinical studies to the clinical objectives. Based on the specific needs of the investigational product as defined in the TPP, an overall regulatory strategy can then be outlined to meet the regulatory requirements for the initial first in human clinical trials. During the preclinical phase of development, sponsors may request meetings with FDA to gather feedback on the planned studies and regulatory strategy. To pave the way for PaVe-GT’s first investigational AAV gene therapy lead candidate, AAV9-hPCCA, we sought early feedback from FDA utilizing an INitial Targeted Engagement for Regulatory Advice on CBER/CDER ProducTs (INTERACT) meeting. Here, we elaborate on the value of TPP and INTERACT meeting. The regulatory documents along with templates developed will be released on the PaVe-GT website.

Scientific Focus Area: Clinical Research

This page was last updated on Tuesday, August 6, 2024

Download or preview the program here

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Current Research Festival

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  • 2024
    • General Schedule of Events
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