MilliporeSigma

Analytical Development and Characterization for Adeno-Associated Virus (AAV)

How to determine CQAs for your AAV

Adeno-associated virus (AAV) mediated gene therapies and any precursor starting materials must be well-characterized to understand the relationship between product quality attributes, manufacturing processes, patient safety and efficacy. Given the variety of AAV serotypes and gene therapy strategies intended to treat a myriad of genetic disorders, there is no "one fits all" off-the-shelf packages for AAV characterization. Our phase appropriate analytical development and method validation capabilities provide the product and process knowledge you need to develop a Quality Target Product Profile (QTPP), identify the Critical Quality Attributes (CQAs) for your therapy, aligned with the Chemistry, Manufacturing, and Control (CMC) Information for Human Gene Therapy Investigational New Drug Applications (INDs), US FDA 2020.

When considering all the factors you need to understand about your AAV therapy, we provide expert insights specifically on your product quality attributes

Patient Factors

Patient Factors

    Product Attributes

    Product Attributes

      Clinical Outcomes

      Clinical Outcomes

        Technical Markers

        Technical Markers

          Every gene therapy is unique. Leverage the expertise of our scientists to ensure compliance, robustness, and efficiency of methods for your viral vector-mediated gene therapy. We are your partner from development through to commercialization, so your therapy can reach patients quickly and safely.


          SMALL VOLUME AAV TESTING

          One of the biggest challenges to method development for characterizing a novel viral gene therapy is efficiently using the limited representative R&D material available.  With this often-restrained resource, key orthogonal assays to confirm product identity, purity, and potency are essential to understand the molecule and mitigate any potential development risks.

          Our Small Volume AAV testing package, arms you with the ability to learn about multiple CQA’s early in development, using reduced quantities of test material, associated with the following characteristics:

          • Identity & titer by ELISA
          • Identity (& Post-Translation Modifications) by LC-MS
          • Aggregation state & capsid integrity by SEC-MALS
          • Aggregation state by DLS
          • ddPCR for genomic titer

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          ASSAYS THAT DE-RISK DEVELOPMENT AND MANUFACTURING

          To help you de-risk and expedite the product and process development of your AAV therapy, we offer a broad selection of assay packages for various CQA categories and process control points:

          • Identity confirmation of plasmid, bulk harvest, drug substance, and drug product
          • Titer and potency determination of bulk harvest, drug substance, and drug product
          • Purity assessment of plasmid, bulk harvest, drug substance, and drug product
          • Residuals testing in drug substance and drug product

          We create tailored programs to uncover your AAV vector’s attributes.


          Capsid Characterization

          • Identification of AAV serotype by ELISA
          • Detection and identification of a Genomic Region of Interest by qPCR
          • Sequencing of vector genome by NGS
          • Capsid protein molecular weight by LC-MS
          • Peptide mapping and post-translational modifications of capsid protein by LC-MS
          • Capsid integrity by SEC-MALS
          • AAV particle titer by ELISA or HPLC
          • Genomic titer by ddPCR
          • Infectious titer: TCID50
          • Potency assessment of r-AAV expressing protein
          • AAV vector purity by SDS-PAGE or CE-SDS
          • Empty/full capsid ratio by Analytical Ultracentrifugation (AUC) or AEX-HPLC
          • AAV capsid aggregation by DLS or SEC
          • Vector aggregates by DLS
          • Plasmid supercoiling
          • Residual host cell DNA
          • Residual host cell proteins
          • Residual AAV affinity ligand by ELISA
          • Residual transfection reagents (e.g. PEI), solvents, and process reagents
          • Residual bovine serum albumin (BSA)
          • pH
          • Osmolality
          • Total protein concentration
          • Appearance
          • Particulates
          • Packaging integrity
          • Extractable volume
          • Bioburden
          • Adventitious viral agents
          • Mycoplasma
          • Endotoxin
          • Sterility
          • Replication competent AAV (rcAAV)


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