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Related Concept Videos

Gene Therapy00:59

Gene Therapy

Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be inserted. The...
Gene Therapy00:59

Gene Therapy

Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be inserted. The...

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Related Experiment Video

Updated: Jun 21, 2026

A Validatable Droplet Digital Polymerase Chain Reaction Assay for the Detection of Adeno-Associated Viral Vectors in Bioshedding Studies of Tears
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A Validatable Droplet Digital Polymerase Chain Reaction Assay for the Detection of Adeno-Associated Viral Vectors in Bioshedding Studies of Tears

Published on: July 14, 2023

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

Rodica Stan1, Richa Madan Lomash1, Oleg A Shchelochkov2

  • 1Therapeutic Development Branch, Division of Preclinical Innovation, National Center for Advancing Translational Sciences, NIH, Rockville, Maryland, USA.

Human Gene Therapy
|June 19, 2026
PubMed
Summary

The Platform Vector-Gene Therapy program streamlines adeno-associated virus (AAV) gene therapy development for rare diseases. Key learnings from FDA meetings accelerate regulatory pathways for AAV9-hPCCA, benefiting future gene therapy products.

Keywords:
AAV9FDA type B meetingFDA type C meetingPCCAPaVe-GTgene therapypre-IND meetingpropionic acidemiarare diseases

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Last Updated: Jun 21, 2026

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Published on: October 11, 2024

Area of Science:

  • Gene Therapy
  • Rare Diseases
  • Regulatory Science

Background:

  • The National Institutes of Health (NIH) Platform Vector-Gene Therapy (PaVe-GT) program develops adeno-associated virus (AAV) gene therapies for rare monogenic diseases.
  • A platform-based approach enhances efficiency in preclinical, clinical, and regulatory processes to accelerate trial initiation.

Purpose of the Study:

  • To share scientific and regulatory learnings from the development of AAV9-hPCCA for propionic acidemia.
  • To outline strategies and lessons learned from Food and Drug Administration (FDA) meetings (INTERACT, pre-IND, Type C) for AAV gene therapy development.

Main Methods:

  • Engagement with the FDA through INTERACT, pre-IND, and Type C meetings for AAV9-hPCCA.
  • Discussion of regulatory topics including animal studies, toxicology, manufacturing, and clinical trial design.
  • Analysis of FDA feedback to inform product development and regulatory strategy.

Main Results:

  • Single rodent species pharmacology/toxicology studies suffice for initial IND filings.
  • FDA feedback aids product quality enhancement and potency assay development.
  • Biomarker data from natural history and Phase 1/2 studies support surrogate endpoints.
  • Phase 1/2 trial data can support future license applications.

Conclusions:

  • The PaVe-GT program's platform approach and regulatory engagement provide valuable insights for rare disease gene therapy development.
  • Sharing regulatory learnings and resources accelerates the path to clinical application for AAV gene therapies.
  • The program's experiences with AAV9-hPCCA offer a model for efficient regulatory navigation in gene therapy.