Genome-titer design
Review sample pretreatment, genome release, target location, matrix effects, reference materials and reporting conventions.
Scientific advisory / AAV
AAV analytical characterization
Build a connected picture of genome, capsid and biological function—with orthogonal evidence for each.
The analytical question
Define what the result means.
AAV quality cannot be summarized by one molecular result. We help develop a characterization strategy that distinguishes packaged-genome attributes from capsid content, impurities and biological activity. Assay selection takes account of the construct, capsid, sample matrix, stage of development and intended decision.
- Genome titer and sequenceqPCR or dPCR can quantify a selected genome target. Complementary approaches are needed to examine sequence identity, truncations, rearrangements or other genome heterogeneity.
- Capsid concentration and contentCapsid-associated measurements address different attributes from PCR. Full, intermediate and empty populations require a suitable physical characterization strategy and careful interpretation.
- Infectivity and potencyCell entry, transgene expression and relevant biological activity are distinct endpoints. A product-specific potency strategy should connect the chosen assay to the intended biological function.
Potential workstreams
How we can help.
The final scope is agreed for the product, development stage and scientific decision.
Genome integrity and heterogeneity
Plan complementary molecular and sequencing readouts; identify regions or molecular structures that an individual assay may not resolve.
Orthogonal capsid assessment
Map candidate physicochemical and capsid methods to the intended attribute and the limitations of each measurement.
Impurity characterization
Assess approaches for residual host-cell and plasmid nucleic acids alongside other relevant process and product impurities.
Potency strategy
Define the relationship between a proposed functional readout, mechanism-relevant activity and the evidence needed to support its use.
Comparability and stability
Select a set of complementary assays and prospective interpretation criteria for process changes and stability studies.
Consulting deliverables
What you receive.
Depending on the agreed engagement, outputs can include:
- An AAV attribute-to-method matrix
- A genome-titer and pretreatment assessment
- An orthogonal characterization study brief
- A potency-strategy and evidence-gap assessment
- A comparability or stability study outline
These are proposed advisory outputs, not claims of completed experiments or validated performance. Laboratory execution and provider responsibilities are scoped separately.
Scientific boundaries
A positive amplicon does not establish a complete intact genome. A genome-to-capsid ratio is not automatically an exact percentage of functional full capsids. Physical fullness alone does not prove correct sequence or biological activity.
Common questions
Does dPCR measure full and empty capsids?
dPCR measures assay-detectable nucleic acid targets. It does not directly classify individual capsids by physical content. Capsid and genome results can be interpreted together only with appropriate assumptions and supporting evidence.
Can NGS establish that every packaged genome is correct?
No. Sequencing can characterize the molecules represented by its preparation and analysis. Coverage, recovery, library bias and resolution constrain the conclusions; consensus identity does not prove that every molecule is intact.
Selected references
These sources inform the scientific principles. They do not establish company credentials or endorse Genometrion.
- FDA — CMC information for human gene therapy INDs
- McColl-Carboni et al. — AAV capsid, genome and function characterization
- ICH Q14 — Analytical procedure development
Editorial review: 31 October 2026.
Related services
Start with the question
Complex therapies.
Clearer analytical decisions.
Define the scientific question behind your project. We can discuss the evidence, methods and practical next steps.
Scientific enquiries
Let’s talk about your project.
A brief, nonconfidential outline is a useful starting point.
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