Adeno-associated viruses (AAVs) are commonly used in gene therapy as vectors to deliver targeted treatments for various genetic conditions and diseases.
An adeno-associated virus is a small, simple, non-enveloped parvovirus comprising a single-stranded DNA molecule enclosed in a protein capsid. AAVs can be used to treat monogenic and ocular diseases. These vectors have become central to clinical trials of treatments for deficiencies such as hemophilia and thalassemia.
The use of an adeno-associated virus for the targeted delivery of gene therapies has the benefits of low immunogenicity, non-pathogenicity, and low likelihood of an inflammatory response in patients. Furthermore, long-term gene expression is retained using this type of viral vector.
This article will briefly explore the importance of quantitation in AAV process development and how Gator Bio’s innovative solutions can aid the characterization of these promising viral vectors.
The importance of quantitation
An important element of adeno-associated virus development is the genome and capsid titer quantitation. This is vital for ensuring efficacious and safe dosing, as problems, such as increased inflammatory responses, can occur if the vector dose is too high.
PCR-based methods are commonly used for AAV quantitation but suffer significant drawbacks. They cannot provide viral particle integrity loss information and can be contaminated by salts, proteins, and plasmid DNAs, potentially providing inaccurate quantitation.
ELISA and other established immunoassays, such as Dot blot for capsid titer quantitation, are labor-intensive and time-consuming. These methods are insufficient for AAV process development and manufacturing.
Gator Bio’s solutions for accurate quantitation
Gator® Plus from Gator Bio is a cost-effective plug-n-play bioanalytical tool for high-throughput serotype quantitation. AAV titer can be performed in a parallelized, automated process, significantly benefiting researchers and clinicians.
Based on Biolayer Interferometry (BLI) technology, the system utilizes CaptureSelect AAVX nanobody probes. These probes bind and quantitate AAV capsid serotypes with a dynamic range of 1×109 – 1×1013 vp/mL.
The assay can be used for high-throughput quantitation of AAVs at all stages of process development, from harvesting to final quality control. The probes used in Gator® Plus are compatible with numerous process development matrices. Eight parallel channels can process up to 96 samples in less than 30 minutes.
The probes are reusable up to 10 times without any binding loss, saving process time and operating costs. This impressive regenerative performance is a central benefit of Gator Plus® for researchers.
Easy data analysis and sample AAV concentration measurement is facilitated by integration with GatorOne® software. This software integration enhances process efficiency as the time needed to repeatedly generate standard curves is saved.
Contact the experts
Efficient, high-throughput adeno-associated virus titer is essential for the development of safe and efficacious viral gene therapies. Contact the experts at Gator Bio to discuss the company’s quantitation solutions today.
References and further reading
- Martinez-Fernandez de la Camara, C. et al. Genes 2021, 12, 601. https://doi.org/10.3390/genes12040601
- Shmidt, A.A. et al. Pharmaceuticals 2022, 15, 23. https://doi.org/10.3390/ph15010023
- Cui, M. et al. Molecules 2019, 24, 3973; doi:10.3390/molecules24213973
- https://www.gatorbio.com/wp-content/uploads/2022/04/AAVX-vs-Progen-App-Note.pdf
About Gator Bio, Inc.
Gator Bio is a world-leading biosensor company headquartered in Palo Alto, CA. At Gator Bio, we provide researchers the tools and instrumentation to advance their research. From antibody engineering to small molecule drug discovery to basic research, Gator Bio can be used to bring meaning to the unknown. From the original inventors of label-free biolayer interferometry (BLI), Gator Bio provides the next generation of BLI technology.
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