GMP AAV
As cell and gene therapy (CGT) landscape expands, Adeno-Associated Viruses (AAV) hold great promise in advancing gene therapy treatments. AAV vectors offer unique advantages, such as tissue tropism specificity, relatively low immune response, non-host genome incorporation and a non-disease causing agent.
While great promise, mass production of AAV under serum-free conditions is a substantial bottleneck in AAV commercialization, until now. uBriGene’s independently developed technology platform has enabled large-scale industrial production of multiple batches of GMP AAV under serum-free conditions in suspension cells. Our platform greatly increases viral production and purity while reducing manufacturing costs.
Production Workflow
uBriGene’s AAV preparation platform utilizes serum-free suspension cultures of our proprietary 293XS cell line. Upstream cell cultures can be scaled up to 2000L with downstream AAV titers productions of >1e14 vg/L. All GMP production of AAVs are done in a Grade C+A environment with an absolute Grade A automated filler.
The whole production process from upstream cell culture, plasmid transfection, harvest and clarification and downstream two-step column chromatography, aseptic filling and storage is operated in a closed single-use system.
Production Technique Highlights
Established Production System
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Established serum-free suspension cell culture
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High performing suspension 293XS cells
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High cell density capability for high AAV yeilds
Multiple Serotype Purification
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Established AAV production and purification platforms
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Multiple serotype manufacturing processes
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rAAV2/2, rAAV2/5, rAAV2/8 and rAAV2/9 production and purification
High Efficiency Manufacturing
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Experienced manufacturing team
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Low rate of empty capsid production
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High yield and purity production facilities
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High cost efficiency of manufacturing
Linear Scalability
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Cell culture scalability from 25 to 2000L single-use bioreactors
AAV Packaging
Adeno-associated virus (AAV) has been a popular viral vector for gene therapy due to its versatile tropism, its non-pathogenicity and low immunogenicity, as well as its ability to transduce dividing and non-dividing cells.
AAV Project Production Cycle
With extensive vial manufacturing and release experience, uBriGene can provide complete clinical-grade viral production services. From cell bank construction, technology, methodology to production and stability testing, our expertise and facilities are here to meet clinical project needs. uBriGene can also provide GMP production documentation, batch inspection records and related certification to assist in IND application filing.
Quality Control and Assurance
With our one-stop shop solution to our client’s cell and gene therapy needs, we provide the highest quality QA and QC support. Our dedicated and experienced quality teams assist our clients in developing testing methods to meet even the most stringent requirements of GMP projects.
Comparison with Other Viruses
Characteristic | AAV | Adenovirus | Lentivirus |
|---|---|---|---|
Origin | AAV-2 | Adenovirus type 5 | HIV-1 |
Genome | 4.8 kb (ssDNA) | 36 kb (dsDNA) | 9 kb (ssRNA) |
Packaging capacity | 4.7 kb | 7.5 kb | 9 kb |
Infection | Most dividing and non-dividing cells | Most dividing and non-dividing cells | Most dividing and non-dividing cells |
Transduction efficiency | Moderate | High | Moderate |
Integration | Non-integrating | Non-integrating | Integrating |
Expression | Transient or stable | Transient | Stable |
Immunogenicity | Very low | High | Low |
Biosafety | BSL-1 | BSL-2 | BSL-2 |
Advantages | High transduction efficiency, Wide range of applications, Stable hereditary | High transduction efficiency, Large capacity, High titer | Low immunogenicity, High safety, High specificity |