Adeno-Associated Virus in Gene Therapy
Considerable advancement in gene editing and gene delivery technologies in recent years has led to a rapid development of viable therapeutics based on gene therapy strategies. There are gene therapy drug candidates in clinical trials for a broad range of disorders, including cancers, hematological conditions, ocular diseases, neuromuscular disease, immunodeficiencies, and rare or inherited disorders. A significant percentage of these gene therapies are based on the use of the adeno-associated viral (AAV) vector, one of the most promising vectors for gene delivery.
Despite rapid growth of the gene therapy market, manufacturing of these therapeutics continues to be a challenge due to a complex production process. At Sartorius, we strive to simplify and accelerate progress in life science and bioprocessing. To this end, the Octet® BLI platform emerges as a pioneering solution, designed to optimize, and streamline viral vector development, manufacturing, and quality control, delivering results in as little as 15 minutes.
Watch the Video: Rapid Quantitation of AAV Capsids in Bioprocess Development
ELISA has traditionally been used to measure AAV viral capsid and is often considered the method of choice for AAV titer measurement. Octet® AAVX assays provide a viable solution to some of the limitations associated with ELISA, such as the thin data sets provided by end‑point assays, as well as the considerable time and effort to perform tasks. The Octet® BLI system design allows for rapid assay setup and analysis. The AAVX biosensor quantitation assay consists of a single step, and AAV titers can be measured in as little as 15 minutes. Another key benefit is that data analysis is displayed in real time, providing the ability to monitor and optimize every assay step.
Importance of AAV Capsid Titer
Adeno-associated viral vector is widely used for the delivery of genetic material in gene therapy, and AAV capsid titer is a critical quality attribute that must be monitored throughout process development and production of AAVs. The development and production of AAVs at an industrial scale requires efficient methods for accurate quantification of viral capsids.
Traditional techniques such as ELISA, HPLC, and ddPCR that are used for AAV capsid titer measurement are time-consuming and labor intensive. For example, AAV titer measurement using ELISA requires six steps and can take anywhere
from three to four hours to complete. ddPCR can take up to six hours. In contrast, using the Octet® BLI platform similar results can be achieved in just one step and
in as little as 15 minutes.
On-Demand Webinar: A New-Wave Biosensor Approach for Direct, Fast, and High-Throughput AAV Process Development
This webinar will highlight REGENXBIO’s experience in finding a high-throughput technology for AAV capsid concentration quantification including comparisons between Octet® AAVX Biosensors and other analytical methods. They will also present an assessment on reliability of % full capsid data generated using various methods including Octet® AAVX Biosensors, as well as case studies making use of the quantification information generated from this technology.
Product Highlights
Octet® BLI Platform
For over 20 years, we have dependably supported our customers by creating established fluidic-free Bio-Layer Interferometry (BLI) platforms, consumables, and software. These products are trusted by scientists worldwide to support their bioanalytical needs for protein interaction analysis spanning from drug discovery to antibody engineering to bioprocessing and quality control (QC).
- Powered by 20+ years of groundbreaking scientific research
- More than 3,500 instrument installs worldwide
- Octet® users invest in genuine BLI performance
- Every Octet® system comes with superior scientific and technical support
- Count on 150 years of Sartorius’ established excellence
Octet® AAVX Biosensors
Rapid Quantitation of AAV Capsids for Various Serotypes for Bioprocess Development and Quality Control in Gene Therapy
- Rapid, direct, and label-free quantitation of AAV capsids in crude and purified samples
- Specificity to various AAV serotypes, including AAV1 – AAV9 and AAVrh10
- Direct quantitation of AAV capsids in the dynamic range of 8.5E8 – 1.0E13 vp/mL
- Efficient and cost-effective regeneration for biosensor reuse up to 10 - 20 times
Featured Content
Industry Evaluation of Octet® AAVX Biosensors for Fast AAV Titers
Researchers at BridgeBio Pharma Inc. and REGENXBIO Inc. recently conducted a thorough evaluation of the new Octet® AAVX Biosensors. The study encompassed a range of AAV serotypes, from various stages in their AAV bioprocess workflow, and included both native (wild-type) and recombinant variants. The accurate quantification of AAV capsid concentrations is a critical step in the development of these vectors. However, traditional methods are slow, labor-intensive, and lack of consistency.
Unveiling the Secrets of Adeno-Associated Virus: Novel High-Throughput Approaches for the Quantification of Multiple Serotypes
This study evaluates analytical methods for determining AAV vector titers, focusing on genome and capsid quantification. It compares traditional assays with advanced techniques, finding that bio-layer interferometry yields comparable capsid titers to ELISA but with increased efficiency and a broader measurement range. The research highlights that employing advanced methods can lead to quicker, more reliable results, and can increase sample throughput while decreasing hands-on lab time.
