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Updated: Jul 5, 2026
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Production of Adeno-Associated Virus Vectors in Cell Stacks for Preclinical Studies in Large Animal Models
Published on: June 30, 2021
Manufacturing and characterizing AAV-based vectors for use in clinical studies.
1Center for Cellular and Molecular Therapeutics, Children's Hospital of Philadelphia, Philadelphia, PA, USA. wrightf@email.chop.edu
Gene Therapy
|April 18, 2008
Summary
Manufacturing recombinant adeno-associated virus (AAV) vectors for gene therapy faces challenges. This review covers Good Manufacturing Practice (GMP) considerations for clinical-grade AAV production.
Area of Science:
- Biotechnology
- Molecular Biology
- Gene Therapy
Background:
- Recombinant adeno-associated virus (AAV) vectors are promising for human gene therapy.
- Clinical translation of AAV-based therapies is hindered by manufacturing and certification hurdles.
Purpose of the Study:
- To review Good Manufacturing Practice (GMP) relevant to AAV vector production.
- To highlight specific considerations and challenges in manufacturing clinical-grade AAV vectors.
Main Methods:
- Literature review of current Good Manufacturing Practice guidelines.
- Analysis of challenges specific to recombinant AAV manufacturing.
Main Results:
- GMP compliance is critical for AAV vector safety and efficacy.
- Key challenges include process scalability, analytical characterization, and regulatory compliance.
Conclusions:
- Addressing GMP challenges is essential for advancing AAV gene therapies to clinical use.
- Standardized manufacturing processes will facilitate the reliable production of therapeutic AAV vectors.
