Gene Therapy Development | Sartorius

Gene Therapy Development

Reduce Time, Effort and Cost in Bioprocessing for Cell and Gene Therapy

Cell and gene therapy (CGT) is a new and promising immunotherapy for patients suffering from certain types of cancer. CGT is among the treatments of Advanced Therapy Medicinal Products (ATMP) transforming how biopharma companies treat and potentially cure certain diseases.

Genes are used to treat, prevent or cure a disease or medical disorder. Often, gene therapy works by adding new copies of a gene that is broken, or by replacing a defective or missing gene in a patient’s cells with a healthy version. With adeno-associated viruses (AAVs) - the viral vector of choice for in vivo treatment - along with a growing interest in gene therapies, there is an increasing need for cost-effective vector manufacturing technologies and viral vector production platforms for human gene therapy products.

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Rapid Quantitation of AAV Capsids in Bioprocess Development for Gene Therapy Using Octet®️ BLI

Rapid Quantitation of AAV Capsids in Bioprocess Development for Gene Therapy Using Octet®️ BLI

Adeno-associated virus (AAV), with low immunogenicity and toxicity, has become one of the most promising viral vectors used for delivery of genetic material in gene therapy. AAV vector development and production at industrial scale requires an efficient method for accurate quantitation of viral capsids in both upstream and downstream bioprocessing.

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. This video compares the Octet® AAVX biosensor workflow to that of ELISA at various stages in bioprocess development.

Featured Applications

Rapid and Direct Quantitation of AAV Capsids

To meet increased throughput and safety demands, scientists at BridgeBio Pharma, Inc. and REGENXBIO Inc. are making use of the novel approach of AAV capsid titer quantification with label-free biosensor assays, which offers a robust alternative to conventional ELISA approaches.

Octet® BLI instruments are eliminating hands-on steps that come with running a traditional immunoassay. Time and human error are significantly decreased, both factors that adversely impact assay reproducibility and team productivity.

Read Application Note

Fast Measurement of AAV Serotypes in Crude and Purified Samples

A rapid and robust method for AAV capsid quantitation is essential for AAV bioprocess development and manufacturing. Conventional ELISA, HPLC and ddPCR methods for AAV titer measurement are time-consuming and labor intensive.

The Octet® Bio-Layer Interferometry (BLI) Platform - using the Octet® AAVX Biosensors - provides a label-free approach with a broad dynamic range from 8.5 x 108 to 1.0 x 1013 vp/mL for all serotypes tested. It also shows high precision and accuracy for capsid quantitation of both purified and crude AAV samples and can be regenerated and re-used up to 20 times providing an efficient and cost-effective solution for high-throughput AAV quantitation applications. 

Read Technical Note

Featured Resources

Webinar

A New Wave Biosensor Approach for AAV Process Development

REGENXBIO Inc. shares their experience in finding a high-throughput technique for AAV capsid concentration quantification.

Octet® AAVX Biosensors for Rapid and Direct Quantitation of AAV Capsids
Application Note

Octet® AAVX Biosensors

For Rapid and Direct Quantitation of AAV Capsids

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Application Note

Biomolecular Binding Kinetics Assays on the Octet® Platform

Introducing label-free platforms, the technology, capabilities, and advantages of label-free binding assessment

A compendium for successful BLI and SPR assays
Application Guide

A Compendium for Successful BLI and SPR Assays

A comprehensive guide for designing and performing assays that accurately measure the binding kinetics of biomolecular interactions and analyte concen...

Webinar

Reduce Cycle Times for AAV Quantification

See the Octet® AAVX Biosensor in comparison to an in-house assay on the Octet BLI platform.

Octet® AAVX Biosensors: For Quantitation of AAV Capsids
Datasheet

Octet® AAVX Biosensors: For Quantitation of AAV Capsids

PDF | 727.5 KB
Octet AAVX Biosensors for Quantitation of AAV Capsids
Technical Note

Octet® AAVX Biosensors for Quantitation of AAV Capsids

PDF | 2.0 MB
Octet RH16 with Lab Tech
Application Note

Converting ELISA Assay Into Octet® Quantitation Assay

PDF | 207.5 KB
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Application Guide

Quality Control Of Biotherapeutics Using Octet® Systems

PDF | 1.9 MB

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