The analysis of adeno-associated virus vectors is of prime importance in gene therapy as well as AAV characterization. As a leader in Protheragen's AAV characterization services and having been at the forefront with advanced analytical technologies, Protheragen together emphasizes the value it brings to aid clients in accelerating their programs.
The characterization of AAVs enables these vectors to achieve their full potential as gene therapy tools. Detailed assessment of systematically defined critical quality attributes (CQAs)of biomolecular structures within AAV preparations reveals a great deal concerning their complex architecture, function, and purity. Such understanding aids in formulating AAV therapeutics with minimized risks while conforming to the rigid rules, such as the high-level regulations on advanced therapeutic medicinal products.
Fig.1 Characterization of AAV gene vectors. (Kontogiannis T., et al., 2024)
The relatively low immune response provoked by AAV vectors, along with their potential for integrating into the host genome and providing long-term expression of therapeutic genes, renders them highly suitable for gene therapy. The in vivo behavior of AAVs, as well as additional assessment of design and regulatory-therapeutic quality safety compliance, requires thorough optimization considerations.
Designing AAV vectors involves various challenges, which modern and classical rigorous technology in tailored bespoke methodology custom for different vector properties probing specific challenges apply. Novel bespoke methods tailored to specific vector properties were applied to address specific challenges posed during AAV characterization.
Table 1. Methodologies for characterization of synthetic AAV capsids. (El Andari J., et al., 2021)
| Technology | Pros | Cons |
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Protheragen will further its commitment regarding AAV characterization technology by advancing methods already in place, adapting to new changes quickly, and evolving techniques to serve clients more efficiently.
Genomic Titer Determination
The titer of AAV vectors is measured quantitatively using qPCR and dPCR. This ensures accurate dosing and therapeutic effectiveness.
Genome Integrity Analysis
With our NGS services, we perform deep analysis involving the complete sequencing of the AAV genome, identifying possible alterations or contaminants that may compromise the safety and efficacy of the vector.
Capsid Protein Characterization
Modification verification of AAV capsid proteins is performed through mass spectrometry (MS) to ensure stability and functionality of the vector post-modification.
Genome Integrity Analysis
Safeguarding the integrity and efficacy of a viral vector involves vigilant scrutiny for hazardous mutations or pathogenic impurities. Next-generation sequencing is critical in achieving this goal, hence forming part of our genomic analysis offering.
Recognizing that every project comes with its own set of challenges, Protheragen offers tailored AAV characterization services customized to particular specifications. Be it the creation of new assays, the refinement of previously established methodologies, or the development of reference materials, we collaborate with our clients to guarantee that their unique needs are fully met. Should our prospective clients wish to inquire more about these services, together with their quotations, they are free to reach us for further details and information on the related services offered.
References
Our AAVLinkTM platform is at the forefront of adeno-associated viral (AAV) vector development, leveraging our extensive expertise and state-of-the-art technologies to drive innovation in gene therapy.
