Avance Biosciences
Molecular testing laboratory supporting cell and gene therapy programs with GLP qPCR and ddPCR assays for biodistribution, shedding and vector persistence, plus assay development and validation.
About AAV Biodistribution
Biodistribution studies show where a therapy goes after dosing, how much reaches each tissue and how long it stays. For AAV gene therapies the standard readout is vector genome copies per microgram of DNA, measured by qPCR or ddPCR across a panel of target and off-target tissues, often paired with transgene mRNA or protein to show where the vector is actually expressed. Shedding analysis of blood, urine, feces and saliva usually runs alongside, because regulators ask whether vector leaves the body and for how long.
The same questions apply beyond AAV. Oligonucleotide programs measure tissue drug levels by hybridization ELISA or LC-MS/MS, and cell therapy programs track where administered cells migrate and persist, commonly with qPCR for human-specific DNA sequences in animal tissue or with in vivo imaging. Whatever the modality, agree the tissue panel, time points and assay sensitivity with the CRO before the study starts, because they are hard to add afterward.
Route of administration drives the result. Intravenous AAV concentrates in the liver, while intrathecal, ICV and intra-cisterna magna dosing produce very different distribution across the brain and spinal cord, and small differences in surgical technique or dose volume can change it further. For CNS programs, the skill of the in-life team matters as much as the assay. Our team has hands-on experience with these CNS routes, and it is part of how we judge the labs listed here.
Biodistribution CROs for AAV and Gene Therapy Studies
Showing 6 CROs. Order rotates daily so no lab is permanently listed first.
Cyagen
Animal model company offering custom genetically engineered rodents together with in vivo pharmacology and AAV services, with operations in the US and China.
VectorBuilder
Vector design and manufacturing company that also offers AAV biodistribution profiling services, including barcoded multiplex studies in rodents and NHPs.
Northern Bio (Northern Biomedical Research)
Preclinical CRO in West Michigan specializing in targeted CNS delivery. Long track record supporting intrathecal, intracerebroventricular and cisterna magna dosing, CSF and tissue distribution, pharmacokinetics and…
GenoSafe
French CRO specializing in analytics for gene and cell therapies, including GLP biodistribution, vector shedding and anti-AAV immunogenicity assays.
Biomere
Biomere (a JOINN company) is a preclinical CRO based in Worcester, MA. Services include non-GLP PK/PD, discovery toxicology, surgical services, disease models, and laboratory sciences. Surgical services include…
Questions to ask when choosing an AAV Biodistribution CRO
- Is your vector genome assay qPCR or ddPCR, and has it been qualified in every tissue on our panel?
- What lower limit of quantitation do you achieve per microgram of genomic DNA?
- How do you prevent cross-contamination at necropsy: collection order, dedicated instruments, separate areas?
- Can biodistribution be added to our toxicology study, or does it need a standalone cohort?
- Do you also run shedding, transgene expression and anti-AAV antibody assays, or are those sent out?
- For CNS routes, how do you confirm the dose was delivered where intended?
AAV Biodistribution CRO FAQs
When is a biodistribution study required for a gene therapy?
Regulators expect biodistribution data before first-in-human dosing of most gene therapies. ICH S12 sets out nonclinical biodistribution considerations for gene therapy products, and FDA and EMA guidance on preclinical gene therapy assessment expects distribution and persistence data in a relevant species, usually by the clinical route. It is often built into the toxicology study rather than run separately.
Biodistribution study design guide →
Which tissues are included in an AAV biodistribution study?
A typical panel covers the target tissues, the injection site, the liver, which takes up most intravenous AAV, gonads to assess germline transmission risk, and other major organs such as heart, lung, kidney, spleen, brain and spinal cord. Blood and excreta are sampled for shedding. Add tissues where the capsid is known to accumulate or where toxicology findings appear.
How is vector biodistribution measured?
Vector genome copies are measured by qPCR or ddPCR and reported per microgram of genomic DNA, using an assay qualified in each tissue matrix. Transgene mRNA (RT-qPCR) or protein (immunohistochemistry, ELISA or mass spectrometry) show where the vector is expressed, not just present. Strict necropsy procedures are needed to prevent cross-contamination between tissues.