Manufacturing Clinical Grade Recombinant Adeno-Associated Virus Using Invertebrate Cell Lines
Abstract
Recombinant adeno-associated virus (rAAV) vectors are proving to be a reliable gene transfer system for several clinical applications, with an increasing body of evidence supporting safety and efficacy. Realizing the clinical and commercial potential of rAAV depends on a reliable source of high-quality, well-characterized rAAV lots. This requirement has been very challenging to achieve due to limits of manufacturing platforms, lot-to-lot variability, or differences in the rigor applied to quality-control assays. In addition to reliable, high-quality vectors, limited quantities of rAAV have hampered clinical development and discouraged investigations into applications that require large therapeutic doses or quantities needed to treat large patient populations. A minimal number of vector production runs should be sufficient to support all phases of clinical development, including non-clinical, pharmacological, and toxicological studies, as well as clinical studies and commercial supply. The production platform using the Sf9 invertebrate cell line has emerged as a scalable and economical source of rAAV. Access to larger quantities of rAAV has now enabled evaluation of gene therapeutics for diseases that require large doses per patient or diseases with large patient populations. The only licensed rAAV product, Glybera, was produced in Sf9 cells, and other rAAV products are in clinical trials in the United States and Europe. The development of the Sf9 rAAV genetics, processes, and overview of the current system are described.
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Different factors, such as the length and type of the vector genome, contribute to variability in the overall ratio of capsids without the intended vector genome.
“Different factors, such as the length and type of the vector genome (i.e., single stranded or self-complementary), contribute to the variability in the overall ratio of capsids without the intended vector genome.19,29”
From Analytical methods for process and product characterization of recombinant adeno-associated virus-based gene therapies (2021) · cites this paper as [19]
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- Authors
- Kotin, R. M., & Snyder, R. O.
- Affiliations
- University of Massachusetts Chan Medical School (US)
- Brammer Bio, Alachua, Florida.
- ORCID
- Kotin, Robert M.
- Venue
- Human Gene Therapy
- Publisher
- SAGE Publications
- Published
- 2017-04
- Volume
- 28
- Issue
- 4
- Pages
- 350-360
- ISSN
- 1043-0342 (ISSN-L)
- 1557-7422
- OpenAlex
- W2609099920
- Cited by
- 59
- References
- 87
- Citations by year
- 2026: 1 · 2025: 4 · 2024: 2 · 2023: 8 · 2022: 4 · 2021: 11 · 2020: 9 · 2019: 3 · 2018: 13 · 2017: 4
- FWCI
- 5.25
- Type
- journal article
- Language
- en
- Open access
- closed
- Locations
- Human Gene Therapy (publishedVersion)
- PubMed (publishedVersion)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › Viral Infectious Diseases and Gene Expression in Insects
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Keywords
- recombinant adeno-associated virus, insect cell expression, gene therapy vectors, lot-to-lot variation
- MeSH terms
- Dependovirus, Animals, Cell Line, Genetic Vectors, Humans, Invertebrates, Genetic Therapy
About these checks
Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.
Statistics and arithmetic are recomputed, not judged.
Confidence bands are shown only for fields calibrated on our annotated test set. None is calibrated yet, so no band is shown.
What counts as a claim ›How we measure confidence ›How we check this ›
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