L-2230.pdf
Powering Gene Therapy Breakthroughs From Discovery to Delivery With Streamlined Automated Protein Analytics
Analytical Instruments Gene Therapy CQAs
Powerful Analytical Tools
Capillary Electrophoresis (CE) Instruments Characterize viral vectors and assess vector purity and stability with Maurice™ Platforms, our fully integrated capillary electrophoresis (CE) systems that enable rapid analysis of charge and size heterogeneity and charge isoform fractionation.
Maurice and MauriceFlex Scan the QR Code or Visit: bio-techne.com/maurice
Simple Plex Simple Plex™ assays on the fully automated Ella™ platform offer a viable alternative to traditional plate-based ELISAs for rapid multiplex analysis, with enhanced precision, reproducibility, and wider dynamic ranges.
Simple Plex Ella Automated Immunoassays Scan the QR Code or Visit: bio-techne.com/simple-plex
Simple Western Simple Western™ Technology provides analytical-grade protein expression quantification in a multi-attribute platform approach, using just 3 µL of cell or tissue lysate sample.
Simple Western Technology Scan the QR Code or Visit: bio-techne.com/simplewestern
Our assortment of hands- free platforms provide you with consistent, high-quality data by automating sample handling and processing.
Reproducible, Trusted Data
Save precious AAV and Lentivirus samples by leveraging the sensitivity of ProteinSimple platforms. Get the data you need with as little as 3 µL of sample volume.
Stretch Your Samples Further
Be confident in your method performance and regulatory compliance by seamlessly transferring analytical methods between labs and across phases from discovery to manufacturing.
Easy Method Transfer
Generate 21 CFR Part 11 compliant data for streamlined preparation of CMC packages for regulatory submissions.
Data Digitalization
These platforms easily fit into your current workflows and adapt to your changing needs. No matter what throughput you require, results will be robust and reproducible—whether you're scaling up or scaling out.
Scale When You Need To
Get reproducible data faster with 90-minute ELISAs, 3-hour Western Analysis or 15-minute icIEF—all with hands-free operation.
Get Results Faster
Where Innovation Meets Simplicity
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Addressing the Core Components of Quality from Discovery to Delivery
Stability Analysis • Product stability
• Aggregate formation
Identity Do I have the right virus in this product?
Are the VP ratios optimized to maximize infectivity?
Key Question
Viral type/subtype
Capsid protein ratio (VP ratios)
Attribute Detail
✓
✓
✓
✓
Simple Western
Simple Plex
Maurice
Stability Is my viral product stable? Product stability ✓
Purity Are there any protein contaminants in my product?
Can you estimate the empty/full status of my sample?
Process-related impurities
Host cell-related impurities
Capsid content (Empty/Full)
✓
✓
✓
✓
✓
✓
✓
✓
Potency How many viral particles do I have?
How well is my virus working?
How much protein is expressed after treatment?
Physical viral titer
Functional viral titer
Protein expression potency
✓
✓
✓
✓
Purity Analysis • Process-related impurities
• Host cell-related impurities
• Empty/full ratios
Potency Analysis • Physical & functional viral titer
• Transgene expression
Identity Analysis • In-process monitoring
• Serotype analysis
• Capsid protein ratios
Cutting Edge Analytical Technologies for Your Peace of Mind
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Validating viral identity is essential when selecting the right vector for targeted gene delivery. Different AAV serotypes vary in tropism and immunogenicity, and many individuals may have pre-existing immunity to certain serotypes.
As your workflows scale, traditional methods like SDS-PAGE, Western blotting, and mass spectrometry
Potency is one of the most critical quality attributes (CQAs) in gene therapy analysis—making reliable, high-performance assays essential.
Automated platforms for protein quantitation deliver faster results, greater reproducibility, and improved sample efficiency compared to traditional methods.
• In-process monitoring
• Serotype analysis
• Capsid protein ratios
Identity Confirmation Potency
Crude Sample Analysis: Simple Western For measuring complex samples, such as cell and tissue lysates, Simple Western provides reproducible AAV quantitation with 3 µL of sample required per well.
Supernatants, Purified Virus Preparations: Simple Plex Simple Plex assays on the Ella platform offer a fast, sensitive, and reproducible method for reliable viral capsid measurement. Simple Plex assays are available to quantify the viral titer of both lentivirus and adeno- associated virus capsids.
Pure Sample Analysis: Maurice For >70% pure samples, Maurice offers direct, highly sensitive measurements of capsid proteins, enabling one-day method development.
Crude Sample Analysis: Simple Western Simple Western technology provides fit-for-purpose protein expression potency assays, enabling efficient, simple, and inexpensive upstream and downstream process development.
Recommended Recommended
can become time-consuming bottlenecks due to their low throughput.
Switching to an automated method can streamline your process, providing high-resolution, reproducible data while using smaller sample volumes.
Identity is provided by an anti-VP1/2/3 antibody (orange), and purity by total protein (blue).
Assays use industry leading antibodies and correlate to ELISA. Detect only intact capsids with dynamic range of 3.6 logs.
Stacked profiles of four different AAV serotypes analyzed with the Turbo CE-SDS cartridge. Maurice CE-SDS assays identify different AAV serotypes.
Fit-for-purpose transgene protein expression with over 6,900 validated antibodies. Screen up to 96-samples in a little as 3 hours.
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In-process monitoring of AAV serotypes with Simple Western
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Physical viral titer with Simple Plex F I G U R E // 02
Serotype analysis with CE-SDS F I G U R E // 04
Protein expression potency with Simple Western
Viral Vector
12 40
C he
m ilu m in es ce
nc e P1
C hem
ilum inescence P2
0
100000
50000
0
100000
50000
0
100000
50000
0
100000
50000
MW (kDa)
30000 20000 10000 0
30000 20000 10000 0
30000 20000 10000 0
30000 20000 10000 0
66 116 230
Total Protein anti-VP1/2/3
AAV4
AAV7
VP3
VP3
VP2 VP1
AAV9
AAV1
VP2 VP1
Aggregate
0.8 1
Ab so rb an
ce
0
10
0
10
0
10
0
10
Relative Migration Time
Turbo CE-SDS VP3 IS
VP2VP1
1.2 1.4 1.6 1.8 2
AAV6
AAV8
AAV9
AAV1
0.05
Si gn
al (R
FU )
0.05
0.5
5
50
LLOQ
500
5000
Concentration (pg/mL) 0.5 5 50 500 5000 50000 500000
ULOQ
3
G FP
N or m al iz ed
A re a (lo
g)
2
4
6
8
AAV8 MOI (log vg/cell) 4 5 6 7
Lot 1 (Control) Lot 1 (Stressed) Lot 2 (Control) Lot 2 (Stressed)
Streamline assay transfer across teams and sites, accelerating development timelines and supporting consistent data generation at every stage.
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Empty or partially filled AAV particles can compromise the purity and safety of gene therapy products. Traditional methods, such as analytical ultracentrifugation (AUC) and ELISAs, are often unreliable, lack sensitivity, and are difficult to scale for manufacturing.
Transitioning to an automated solution provides consistent and sensitive quantification of empty/full capsid ratios, with the scalability needed to support AAV manufacturing workflows.
Stability assessment is essential for ensuring that AAV vector particles retain their structural and functional integrity over time, helping to preserve potency, safety, and efficacy.
Purity Stability
Upstream Analysis: Simple Western For analysis of unpurified samples, Simple Western enables automated AAV empty/full ratio measurements in <6 hours.
Downstream Analysis: Maurice Save time, labor, and sample compared to AUC and get critical capsid content information with icIEF and CE-SDS using Maurice.
Host Cell Impurities: Simple Plex Simple Plex assays on the Ella platform offer a fast, sensitive, and reproducible method for reliable host cell impurity assessment.
Recommended
AAV Product Stability: Maurice Get high resolution AAV vector stability data with CVs <5% without dependence on particle size, using icIEF with Maurice.
Recommended
Detecting and quantifying residual proteins, such as host cell proteins (HCPs) from HEK293 cells or endonucleases from AAV vector preparations, is also critical for ensuring product purity.
High-sensitivity platforms are essential for identifying low levels of impurities that can affect efficacy, alter product quality, and potentially trigger immune responses in gene therapy applications.
Requires only 3 µL sample (~1012 GC/ml) per well. Applicable to multiple serotypes.
An increase in acidic species signifies deamidation, which in turn correlates with the transduction efficiency of AAVs.
Heat-stressed AAV8 shows substantial changes in its charge profile in as little as 7 days.
Low sample input (4x 1011 GC/mL), data in only 8 minutes.
Detect low levels of impurities as well as AAV genome residuals with only 8-minute separation.
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Monitor deamidation of capsid proteins with icIEF
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AAV stability assessment with intact icIEF methods
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Stability profiling of capsid proteins with denatured icIEF methods
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Rapid and sensitive empty/full capsid quantification with Simple Western
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Impurity measurements with Maurice CE-SDS
Pe rc en
ta ge
(% )
0
100
50
% Empty or partially empty % Full
25%
75%
Ab so rb an
ce (m
AU )
4
3
2
1
0
Relative Migration Time 1 1.2 1.4 1.6 1.8 2 2.2 2.4 2.6
Turbo CE-SDSIS VP3
VP2 VP1
Impurities AAV Genome
AAV9-CMV-GFP scAAV9-hSyn-GFP AAV9-CK8-CAS9 scAAV9-CMV-GFP AAV9-CMV-Luciferase
Fl uo
re sc en
ce
pI value
T4W25C
T2W25C
T1W25C
VP3VP3dpI 4.05 pI 8.40T0 1000 2000
0
0 1000 2000
0 1000 2000
0 1000 2000
8.57.56.56 7 85.554.54
5.5 6
Fl uo
re sc en
ce
0 5000
pI 6.5 7
370C at 14 days
370C at 7 days
370C at 3 days
Control 40C
0
5000 0
5000 0
5000
6
Fl uo
re sc en
ce
pI 6.5 7.5
Reference
P2
P1 P3
P4P5
P6
1 Day
3 Days
8 Days
14 Days
7 0
5000
10000
0
5000
10000
0
5000
10000
0
5000
10000
0
5000
10000
0
Switching to a direct detection method offers greater sensitivity in less time, delivering robust stability data that can help streamline regulatory approvals.
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Meet Our Instruments Scan the QR Code or Visit: bio-techne.com/instruments
Powering Your Research with Trusted Analytics
10,000+ Labs Trusting the Technology
4,500+ Citations
100+ Commercial Products Supported
• Automated Systems With Faster Results
• Reproducible Trusted Data
• Data Digitization Compliance
• Stretch Your Samples Further
• Easy Method Transfer
• Scale When You Need It
S P E C I F I C A T I O N S A T A G L A N C E
Platform Specifications
Main Sample Type
Assay Type
Detection Modes
Sample Volume
Assay Development
Detection Range (GC or VP/mL)
Runtime
Quantitative
Size Separation
Virus, cell, and tissue lysates
Western
Chemiluminescence, Fluorescence, Total protein
3 µL
Single antibody– open platform
109 - 1012
3 hours 5 hours with RePlex™
✓
✓
Simple Western
Virus lysates, supernatants, purified virus preparations
Sandwich ELISA
Fluorescence
25 µL
None– fully validated assay kits
106 - 1010
Less than 90 minutes
✓
Simple Plex
Virus preparation (>70% purity)
CE-SDS, icIEF and icIEF Fractionation
Absorbance and native fluorescence
50 µL
No antibody– open platform
1011 - 1014
5-35 minutes with CE-SDS 10-15 minutes with icIEF
✓
✓
Maurice
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