> Cell and Gene Therapy Solutions > Vector Characterization
ProteinSimple’s analytical platforms give you the automation and scalability needed for the development and manufacturing of gene therapy vectors, and low volume requirements help you preserve these precious samples. Platform methods can be seamlessly transferred across labs and project phases—from discovery to manufacturing—giving you consistent vector characterization results from start to finish.
Automated, high-throughput systems let you characterize AAVs in sample volumes as low as 3 µL.
Stability, identity and purity all on one system: Maurice
Safely delivering a gene therapy means ensuring the stability, identity, and purity of your AAV vector. The Maurice system combines imaged capillary isoelectric focusing (icIEF) and CE-SDS analysis, letting you get all of that data with one compact system. Load up to 100 samples in a batch and walk away, Maurice takes care of the rest. Results are ready in 6-25 minutes using sample volumes as low as 50 µL.
Check vector stability and identity with icIEF
Ensure product stability and identity by characterizing the charge heterogeneity of your AAV vectors with icIEF detection on Maurice. View this Application Note to learn more.
Characterize purity for 48 samples in a day with CE-SDS
Get rapid, well-resolved, and reproducible characterization of your AAV capsid purity with CE-SDS detection on Maurice. Automate up to 48 samples in a batch using sample volumes as low as 50 µL. See how in this Application Note.
Innovative Capillary Electrophoresis Techniques for Gene Therapy R&D
Learn how various capillary electrophoresis techniques can be easily implemented along the development path to support gene therapy development. This webinar focuses on Adeno-associated virus (AAV) Empty and Full separation using the imaged cIEF method with the Maurice system. Dr. Wei showcases how imaged cIEF separates an AAV drug substance that could not be separated using other techniques and illustrates the imaged cIEF method development. This webinar features guest speaker, Dr. Wei, Associate Director, Analytical Development, Ultragenyx Pharmaceutical Inc. and Chris Heger, Senior Manager, Applications Science at ProteinSimple. Watch On-Demand
Simplify your AAV and LNP manufacturing characterization
Learn how cIEF and CE-SDS on Maurice are used in a manufacturing setting to assess purity and stability of AAVs and identity of LNPs. Watch Advances in Cell & Gene Therapy Analytics, a webinar featuring John Loughney, Director, Vaccine Analytical Research & Development at Merck and Chris Heger, Senior Manager, Applications Science at ProteinSimple.
Monitor your AAV capsids during product purification with Simple Western
Simple Western can be used to monitor AAV capsid proteins throughout the purification process using Simple Western™ immuno- and total protein assays. You’ll only need 3 μL of sample to get pg-level sensitivity, and automatically analyzed results are ready within 3 hours. Learn how the Cell and Gene Therapy Consortium Catapult uses highly specific PROGEN antibodies with Simple Western assays on Wes to monitor and characterize AAV capsids during product purification in this Application Note.
Identify AAV Aggregates and Contaminants with MFI
Monitoring of aggregates of AAV capsids is important for ensuring stability of the vector, but methods such as light obscuration and microscopy have limitations with their ability to detect translucent aggregates or lack of throughput. Our Micro-Flow Imaging (MFI) systems directly image subvisible particles in solution to provide data for 10 different morphological parameters, allowing you to identify AAV aggregates as well as any contaminants or impurities in your sample.
Micro-Flow Imaging (MFI) monitors the effect of ultrafiltration on the preparation of AAVs
Ultrafiltration is used to concentrate and prepare AAVs in the final formulation buffer. To measure the effect of ultrafiltration on subvisible particle formation, researchers at Voyager Therapeutics relied on MFI to measure the size and concentration of subvisible particles that formed in solution. This is the first study to provide a quantitative understanding of the ultrafiltration behavior of AAVs for gene therapy to guide process development.
- Ultrafiltration behavior of recombinant adeno associated viral vectors used in gene therapy A Arunkumar, N Singh J. Membr. Sci., Feb 2021; 620:118812. doi: 10.1016/j.memsci.2020.118812.
Characterization of the identity and stability of lipid nanoparticle delivery vectors is key to ensuring the safety and efficacy of your therapeutic. Maurice makes measuring the surface charge of these large, complex particles easy. See how researchers at Merck use Maurice during process and formulation development of an LNP-based mRNA vaccine.
- Development of an imaged capillary isoelectric focusing method for characterizing the surface charge of mRNA lipid nanoparticle vaccines
JW Loughney, K Minsker, S Ha, RR Rustandi
Electrophoresis, Sept 2019; (18-19):2602-2609. doi: 10.1002/elps.201900063.
Simplify Your AAV and LNP Manufacturing Characterization
Learn how cIEF and CE-SDS on Maurice is used in a manufacturing setting to assess purity and stability of AAVs and identity of LNPs. Watch Advances in Cell & Gene Therapy Analytics, a webinar featuring John Loughney, Director, Vaccine Analytical Research & Development at Merck and Chris Heger, Senior Manager, Applications Science at ProteinSimple.
High-Quality Western Blotting Results in 3 Hours
Helper-dependent adenoviral (HD-Ad) vectors have a large carrying capacity. In a study conducted by researchers at The Hospital for Sick Children in Toronto, engineered transcription activator-like effector nucleases (TALENs) and donor DNA were packaged together in a single vector to deliver both to target cells with high efficiency. Learn how they used Simple Western assays on Jess instead of traditional Western blots for analysis of TALEN protein expression at different time points post-transduction, and got fully analyzed results for 24 samples in under 5 hrs.
Solutions for AAV Vector Biodistribution Analysis
Get morphology-based AAV vector biodistribution and transgene mRNA expression analysis in intact animal model tissues with RNAscope™ and BaseScope™ assays from ACD. Explore how these assays combine quantitative, molecular measurement, like PCR, with single-cell resolution in the context of intact tissue morphology, and view ACD’s solutions for Gene Therapy/AAV here at acdbio.com/science/applications/research-areas/gene-therapyaav.