GI Partners Acquires CDMO and Cell Solutions
As Rose BioSolutions, the established CDMO and Cell Solutions businesses continue to support the biotechnology ecosystem with cell sourcing capabilities and CDMO services to accelerate your advanced therapy from development to delivery.
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Smoothly navigate your gene therapy production with a proven AAV manufacturing platform
Built on the experience gained through decades of Adeno-Associated Virus (AAV) vector contract development and manufacturing organization (CDMO) experience, our viral vector center of excellence has established an industry-leading platform to streamline the pathway to GMP AAV vector manufacturing.
Minimizing process development requirements, the nAAVigation® AAV production platform provides viral vector gene therapy developers with the capability to reduce a program's timeline to GMP by up to 55%, or fewer than eight months, when compared to traditional AAV manufacturing workflows.
Based on a proprietary, high-productivity HEK293 suspension cell line, amenable to clients’ scale and serotype needs, the AAV production platform approach utilizes in-house plasmid DNA production, an optimized single-use upstream process coupled with robust downstream purification processes, and 100% qualified, in-house analytical capabilities, to enable clients to efficiently scale up while simplifying supply chains.
“By increasing speed and efficiency for viral vector production, nAAVigation will help accomplish our ultimate goal of delivering safe, effective therapies to patients faster.”
Kerstin Dolph, Corporate Senior Vice President, Global Manufacturing, Charles River
Speed
Expedited development and 100% in-house analytics
Experience
Differentiated CDMO and fully integrated testing powerhouse
Predictability
Cost-effective, reliable path to GMP and clinic
Ready to discuss an upcoming AAV manufacturing program?
Explore our Viral Vector CDMO Facility
Specializing in GMP-compliant production of adeno-associated virus (AAV), lentivirus, adenovirus, and retrovirus for gene and gene-modified cell therapies.
Explore the Facility
Choosing a production cell line for AAV manufacturing
Our viral vector CDMO team has developed proprietary HEK293 and 293T producer cell lines for both adherent and suspension culture GMP AAV production methods. Suspension cultures allow for the use of serum-free, chemically defined media, which in turn can allow for a more streamlined production of vectors intended for clinical use and can be grown to larger scales.
Some constructs, however, do not yield virus efficiently when grown in suspension cultures. We will guide your selection of cell line and culture condition based on the serotype of your AAV gene therapy, size of your transgene, and quantity of GMP AAV required.
| AAV Manufacturing Technology | Key Features | Adherent (CellSTACK; HYPERstacks) | Suspension (50-500 L bioreactors) |
|---|---|---|---|
| Helper-free triple transfection | Fastest turnaround time | HEK293, 293T | HEK293-s, 293T-s |
| Helper virus | Easily scales to commercial quantities | HEK293, 293T | HEK293-s, 293T-s |
GMP AAV Production Workflow
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Simplified and secured supply chain
Standardized processes and partnerships with global providers facilitate standardized BOMs, meaning raw materials are on hand on Day 0.
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Integrated plasmid DNA production
- Our plasmid DNA center of excellence enables us to jump-start your viral vector programs as early as possible.
- Off-the-shelf AAV packaging plasmids (RepCap and Helper) in research, High Quality (HQ), and GMP grades are also available to expedite your AAV production.
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Standardized AAV manufacturing process
With proprietary HEK293 and 293T producer cell lines for adherent or suspension culture, we offer large-scale chromatography AAV purification services (IEX, AEX, affinity, SEC, etc.) based on our clients’ preference, material needs, timeline, and long-term development plan.
The nAAVigation® vector platform provides AAV vector gene therapy developers an expedited, cost-effective, and predictive pathway to GMP manufacturing, with the capability to reduce timelines by 55% or in fewer than eight months compared to traditional process development.
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100% in-house QC
Analytical method development and testing capabilities with a range of pre-qualified platform assays, well-versed assay tech-transfer capabilities, and industry-leading assay development services.
Not ready for GMP AAV manufacturing? Explore research grade AAV packaging services.
What’s Next for AAV Production and Gene Therapy?
Join Drs. Jude Samulski and Ramin Baghirzade to explore the biology of AAV production and the steps required to generate clinical grade vectors, the status of AAV clinical trials, and thoughts on key areas of innovation.
Get the ABC of AAV
Concerned about project continuity and looking to reliably tech transfer an existing AAV production program? Explore Modular and Fast Track viral vector tech transfer frameworks.
Frequently Asked Questions (FAQs) About AAV Production Services
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How long does it take to generate clinical grade GMP AAV?
The protocol for clinical grade AAV production involves many steps to ensure the quality and safety of the final product. Altogether, it can take 10-15 months to complete a GMP project.
The AAV Manufacturing Process:
- The first stage of any campaign is process development, in which various parameters involving the source plasmids, producer cell lines, and purification methods are optimized.
- Following this step, procedures must be adapted to the larger scales necessary to generate the increased amounts of material required for clinical trials.
- Next, an engineering run is performed to confirm that the processes and procedures yield the expected amount of virus at the larger scale.
- After the engineering run, a GMP run that fully complies with FDA and EMA guidelines is performed. This is the run that produces the actual material for clinical use. However, before this material can be used in patients, quality control testing must be conducted to confirm the final product’s identity and purity.
- Finally, release testing must also be completed before the final product is deemed ready for use in patients. The release testing confirms sterility and stability of the final product.
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Why include an engineering run for AAV production?
The engineering run is a vital part of any GMP viral vector manufacturing campaign as it allows for the detection of any unforeseen issues that could impact GMP AAV production.
The engineering run uses the exact same procedure as the GMP run but is not performed in the GMP facility (i.e., no regulated air supply and not subject to QA oversight). This run allows for the confirmation that the campaign-specific processes will be successful, and that the expected viral titer will be obtained.
Material produced during the engineering run can be used for in vitro or in vivo testing (or toxicity studies) but cannot be used in patients.
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Can Charles River produce pHelper and Rep/Cap plasmids to support AAV production?
Yes. Our integrated plasmid DNA center of excellence manufactures phase-appropriate custom or off-the-shelf research grade, High Quality (HQ) intermediate grade, and full GMP plasmids.
Explore Off-the-Shelf Rep/Cap and Helper plasmids for AAV packaging
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What is the typical yield for GMP viral vector production?
Because viral vector titer is highly sensitive to the size of the viral genome, the resulting viral vector manufacturing yield will vary depending on the size of the desired insert.
Please contact us for more information about what yield may be obtainable with your insert.