End-To-End AAV Gene Therapy Solutions
Setting the Standard in AAV Gene Therapy, Together
Adeno-associated virus (AAV) gene therapies have potential to transform the way we treat certain diseases. But to deliver them successfully, robust and cost-effective production is critical.
Bring your therapy to market faster and affordably with end-to-end solutions that deliver reproducible performance and scalability with a simplified path to regulatory approvals.
Spotlight on Success: Matica Biotechnology
Scaling AAV8, Together: A Collaborative Path to Process Innovation
As demand for AAV therapies accelerates, developers face growing pressure to build processes that are scalable, adaptable, and compliant. To meet this challenge, Sartorius and Matica Biotechnology collaborated to create and validate a robust AAV8 production platform, demonstrating consistent performance from bench to 50 L scale. Through an integrated, data-driven approach linking upstream and downstream operations, the teams achieved reproducible titers, effective full-to-empty capsid separation, and strong impurity clearance within a flexible, GMP-aligned framework. This collaboration highlights how shared innovation can simplify complexity, accelerate scale-up, and strengthen manufacturing readiness for next-generation gene therapies.
AAV Gene Therapy Workflow
Solutions for Process Development and Manufacturing
Explore AAV gene therapy solutions to simplify progress in every step of your workflow.
Solutions for AAV Gene Therapy
Connected Services
Plasmid Engineering Service offers custom plasmid engineering based on a unique DNA brick approach to enable fast and precise engineering of any plasmids, even the most complex ones with a success rate above 95%
Plasmid Manufacturing Service excels in producing pDNA at high quality with superior yields and supercoiled integrity. Each plasmid DNA produced is provided with regulatory support to ensure compliance for clinical trials and commercial use.
AAV testing services to support AAV manufacturing from R&D to regulatory approval, ensuring speed and capacity with ready-to-use QC testing plans and pre-validaed cGMP assay and specialized high-quality analytics to test the following attributes identiy, purity, potency and safety.
Cornerstone Development Service is designed to support customers in establishing in-house pDNA purification processes at the required scale and that is then seamlessly transferred to each customer site.
Cell culture media Services include a array of media services including benchmarking services, media optimization, media development and media manufacturing-all supported by spent media analytics
Connected Software & Analytics
DOE software Simplified for Life Science. Ready-to-go connectors for Sartorius equipment & Biobrain Supervise to ease implementation. Backed up with a data science team to get the most out of your data.
Multivariate data analysis software that simplifies your data exploration for Process Experts. Ready-to-go connectors for Sartorius equipment and Biobrain Supervise. Backed up with a data science team to get the most out of your data.
A data acquisition, process control & monitoring system that connects Sartorius and 3rd party unit operations. A scalable system can support 1 to 32 process units to enable PD or GMP production with integration to Umetrics® for DOE or advanced process control.
Featured Products & Services
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AAV R&D Solutions
AAV Research and Development Solutions
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FAQs for AAV Gene Therapy
AAV is a small virus less than 30 nm in diameter, containing 5 kb DNA in a capsid with mostly episomal expression. It has different serotypes depending on the capsid's protein expression, supporting targeting of an organ. They are commonly produced using transfection of two to three plasmids to HEK293 — the cells then undergo purification using filtration and chromatogaphy to remove impurities.
Critical Quality Attributes (CQAs) are typically established during a risk assessment and depend on the specific drug being developed. For most drugs, the focus includes product safety, identity, sterility, purity, and potency. In the case of Adeno-Associated Virus (AAV) therapies, measuring potency is challenging. Potency assessments can involve evaluating genomic and capsid titers, as well as transduction efficiency. Ensuring purity is also crucial to avoid side effects; therefore, monitoring impurities such as empty AAV particles, DNA, and protein content is essential.
To increase performance of AAV processes, we offer HEK media and transfection reagents like FectoVIR®-AAV and pPLUS® AAV-Helper. Combined with single-use bioreactors offering the same geometry from 250 mL to 2000 L, we ensure a simplified scale-up.
We also offer solutions for clarification like Sartoclear® depth filters, Hydrosart® cassettes, Sartopore® 2 XLG sterilizing filter, and monolith columns that are scalable from process development to large-scale manufacturing — in single-use and closed formats.
Downstream processes should remove impurities like DNA and HCP to maximize drug safety and efficacy. In AAV production, separating the large amount of empty capsids — up to 80% — is challenging due to their similarity to full capsids. AAV concentration can also cause aggregation and loss due to binding to filter materials, resulting in low overall performance.
At Sartorius, we offer solutions like Sartoclear® depth filters, Hydrosart® cassettes for TFF with low-binding filters, and monolith technology with QA to efficiently remove impurities and empty capsids. Additionally, Recombumin® recombinant human albumin prevents AAV aggregation.