Innovations to Transform Your Gene Therapy Research
Adeno-associated viruses (AAV) have emerged as a promising vector in the field of gene therapy, offering potential breakthroughs in treating a wide range of genetic disorders. Their ability to deliver therapeutic genes with high precision and minimal immune response makes them an attractive option for researchers and clinicians alike. However, the journey from discovery to clinical application is fraught with challenges.
Common R&D workflow hurdles include optimizing vector design, ensuring efficient gene delivery, and overcoming characterization challenges. Additionally, regulatory compliance and scalability remain critical concerns that researchers must navigate to bring these innovative therapies to patients. Despite these challenges, the potential of AAV in transforming disease treatment continues to drive significant advancements in the field.
Sartorius offers industry-leading solutions to ensure efficiency, developability, and scalability throughout the entire AAV gene therapy R&D workflow. Our advanced cell and protein interaction analytical methods deliver faster and more robust results, significantly increasing sample throughput while reducing bench time.
Featured Solutions
Octet® Label-Free Bio-Layer Interferometry (BLI) Detection Systems
Octet® Bio-Layer Interferometry (BLI) systems in combination with the Octet® AAVX Biosensors offer a rapid, label-free, and high-throughput method for quantification of AAV capsids.
- High precision and accuracy for capsid quantitation of both purified and crude AAV samples
- Rapid, and high-throughput approach for determining AAV empty/full ratio in as little as 30 minutes
iQue® High-Throughput Screening (HTS)
Enables the assessment of a range of cellular parameters in a single well, allowing for a streamlined workflow, improved data coherence and a highly informed data set.
- High-throughput screening of AAV vectors
- Quantitative analysis of AAV transduction efficiency
- Detailed insights into cell health and viability
Incucyte® Live-Cell Analysis System
Incucyte® Live-Cell Analysis System enables the monitoring and quantitation of the efficiency and timecourse of gene transfection using GFP/RFP constructs.
- Precise assessment of transduction efficiency
- Improved accuracy
- Reduced manual steps
Accelerated TCID50 Assay Using Live-Cell Analysis Application Note
Lab-Scale Clarification and Purification
Addressing the challenges of isolation, concentration and purification of AAV particles with well-coordinated workflow steps for optimal results.
- Sartoclear Dynamics® Lab for convenient mammalian cell culture harvesting
- Vivaflow® tangential flow filtration (TFF) cassettes and Vivaspin® centrifugal filters for concentration and diafiltration of AAV particles with unparalleled speed and flexibility
- Sartobind® IEX membrane chromatography units for fast capture and empty/full separation, available in a range of ready-for-use formats that minimize set up time and ensure simple, flexible purifications.
- Minisart® and Sartolab® Filtration Units & Systems for particle removal and sterile filtration
Advanced High Throughput Cytometry, Live-Cell Analysis, and Biolayer Interferometry platforms provide data-rich assays and therefore more insights into Critical Quality Attributes (CQAs). Researchers can gain a deeper understanding of AAV vector properties and performance, enabling more efficient optimization and validation processes.
Enhanced reproducibility with complimentary assays
Our innovative, lab-scale filtration and purification solutions such as Sartoclear Dynamics® Lab for mammalian cell culture harvest, Vivaflow® tangential flow filtration (TFF) cassettes and Vivaspin® centrifugal filters for sample concentration and buffer exchange, and Sartobind® IEX Lab membrane chromatography units, provide a holistic solution to ensure high purity levels with reduced complexity. These improvements not only accelerate the purification timeline but also maintain the integrity and quality of AAV vectors.
Featured Resources
Frequently Asked Questions
Common challenges include optimizing vector design, ensuring efficient gene delivery, overcoming characterization hurdles, developability, and scalability issues.
Sartorius offers advanced analytical solutions for comprehensive assessment of critical quality attributes (CQAs) that enhance efficiency and developability of gene therapy candidates
Sartorius offers high-throughput generation and clonal selection platforms like CellCelector and Octet® BLI systems for optimizing AAV titers and productivity, along with tools for monitoring cell health and viability with iQue® HTP Cytometry and Incucyte® Live-Cell Analysis Platforms.
It offers a rapid, high-throughput method that analyzes intact viral capsids without the need for sample purification or enzymatic digestion, completing the assay in as little as 30 minutes.
AAV Empty/Full Ratio Assessment Using the Octet® AAVX Biosensors Technical Note
Sartoclear Dynamics® Lab for cell culture harvesting, Vivaflow® TFF cassettes, Vivaspin® Centrifugal Filters for concentration and buffer exchange, and Sartobind® IEX Lab for purification, which ensure high purity and an efficient end-to-end workflow.