ArticleMolecular therapy. Methods & clinical development2024
Adeno-associated virus perfusion enhanced expression: A commercially scalable, high titer, high quality producer cell line process.
Article in Molecular therapy. Methods & clinical development, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 10 papers.
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Who cites it
10 citing papers in PubMed.
- Comparative Analysis of rAAV Production from Plasmid-Encoded Versus Chromosomally Integrated rAAV Transgene in HEK293 Cells.International journal of molecular sciences · 2026Article
- Manufacture of adeno-associated virus vectors by a novel human-derived cell line HAT and comprehensive evaluation of the vectors.Molecular therapy. Advances · 2026Article
- Enhancing gene therapy vectors manufacturing: CEBPA as a master regulator of rAAV biogenesis.Journal of biological engineering · 2026Article
- Streamlined rAAV HeLaS3 producer cell line generation via GS selection.Scientific reports · 2026Article
- Accelerated Adeno Associated Virus Upstream Process Development From High-Throughput Systems to Clinical Scale.Biotechnology and bioengineering · 2025Article
- Self-silencing adenovirus enables precise infectious titration of recombinant adeno-associated viral vectors.Molecular therapy. Methods & clinical development · 2025Article
- Establishing a scalable perfusion strategy for the manufacture of CAR-T cells in stirred-tank bioreactors using a quality-by-design approach.Bioengineering & translational medicine · 2025Article
- Optimising and adapting perfusion feeds in serum-free medium to intensify CAR-T cell expansion in stirred-tank bioreactors.Frontiers in bioengineering and biotechnology · 2025Article
- Status and future of recombinant adeno-associated virus vector manufacturing.Biotechnology progressReview
- Transcriptomic functional characterization of recombinant adeno-associated virus producing cell line adapted to suspension-growth.Biotechnology progressArticle
Corrections and comments
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Authors and funding
7 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
With safety and efficacy demonstrated over hundreds of clinical trials in the last 30 years, along with at least six recent global marketing authorizations achieved since 2017, recombinant adeno-associated viruses (rAAVs) have been established as the leading therapeutic gene transfer vector for rare, monogenic diseases. Significant advances in manufacturing technology have been made in the last few decades to address challenges with GMP production of rAAV products, although yield, cost, scalability, and quality remain a challenge. With transient transfection processes established as a manufacturing platform for multiple commercial AAV products, there remains significant yield, cost, robustness, and scalability constraints that need to be resolved to enable a reliable supply of rAAV products for global patient access. The development of stable producer cell lines for rAAV products has enabled scalability and, in some cases, improvements in productivity. Herein we describe a novel AAV perfusion-enhanced expression (APEX) process, resulting in higher maximum cell densities in the production bioreactor with a 3- to 6-fold increase in volumetric productivity. This process has been successfully demonstrated across multiple serotypes in large scale cell culture with titers approaching 1 × 10
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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.