Services → Biodistribution

Biodistribution for gene therapy drug development

GLP-accredited qPCR and dPCR assays for precise biodistribution analysis of gene therapy products, designed to meet the regulatory expectations of FDA and EMA.

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BIODISTRIBUTION CRO

Custom biodistribution assays for:

AAV biodistribution

Quantifying AAV vector copy numbers and transgene expression in target and non-target tissues, as well as biofluids, using qPCR and dPCR. GLP or non-GLP compliant services.

CAR-T and cell therapy biodistribution

Tracking and quantifying the biodistribution of therapeutic cells using qPCR and dPCR. GLP or non-GLP compliant services.

mRNA biodistribution

Quantifying the biodistribution, persistence, and clearance of administered mRNA and mRNA vaccines using qPCR and dPCR. GLP or non-GLP compliant services.

siRNA, miRNA and ASO biodistribution

Using TATAA’s Two-Tailed PCR technology, short nucleic acid targets are elongated and amplified with highly sensitive qPCR and dPCR.

A comprehensive guide

Biodistribution assay design and analysis

In vivo biodistribution of gene therapy drugs, mRNA, mRNA vaccine, and RNA therapeutics like siRNA and miRNA poses additional challenges for quantitative PCR assays compared to standard gene expression studies. The low copy number of these molecules demands exceptional assay sensitivity and robustness. Download the guide to learn more.

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Preclinical biodistribution

Assay design and validation
Regulatory guidelines (EMA and FDA)
Challenges and best practices

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GLP for qPCR and dPCR

We are accredited for Good Laboratory Practice (GLP) by the Swedish Board for Accreditation and Conformity Assessment (SWEDAC) for qPCR, dPCR, and molecular biology.

Biodistribution at TATAA

Samples

Injection site(s)
Gonads
Adrenal gland
Brain
Spinal cord (cervical, thoracic, and lumbar)
Liver
Kidney
Lung
Heart
Spleen
Blood

Test items

AAV constructs (encapsidated, in NLPs, and as free DNA)
Transgene mRNA
siRNA
Antisense oligonucleotides (ASOs)
mRNA vaccines

A biodistribution study

A biodistribution study is essential in drug development to assess how a therapeutic agent distributes throughout the body over time. These studies are critical for gene therapies and RNA therapeutics to evaluate efficacy, determine safe dosing, and minimize off-target effects.

In preclinical biodistribution studies, multiple tissues and biofluids are analyzed, including the injection site(s), gonads, adrenal glands, brain, spinal cord (cervical, thoracic, and lumbar), liver, kidneys, lungs, heart, spleen, and blood.

In clinical biodistribution studies, vector persistence is monitored to ensure safety and efficacy.

Biodistribution methods and assays

A biodistribution assay encompasses the entire analytical workflow—from the extraction of the test item from tissues or biofluids to cDNA synthesis during the RT step, quantitative PCR analysis, data normalization, and quality control.

During method development, we optimize each step to ensure accuracy and sensitivity. Method validation further guarantees assay robustness, ensuring it meets predefined criteria for LLOQ, LOQ, MQC, HQC, and ULOQ, providing reliable and reproducible results.

qPCR and dPCR assays for biodistribution

Quantitative PCR (qPCR) and digital PCR (dPCR) are highly sensitive methods for detecting nucleic acids, including AAV vectors and transgene mRNA. With Two-Tailed PCR technology, even short targets such as miRNA, siRNA, and ASOs can be accurately quantified.

We design primers and probes to quantify the target without detecting endogenous genes or transcripts. Whenever possible, at least one primer targets non-natural sequences, such as vector backbones, non-natural exon junctions, promoter regions, endogenous genes, or codon-optimized sequences, ensuring specificity.

To maintain the highest standards of accuracy, RNA spikes are added to check for matrix interference during the RT step, and DNA spikes to monitor interference in qPCR/dPCR analysis.

We optimize our assays to ensure unbiased quantification across different constructs, regardless of modifications, sequence lengths, or structural variations, enabling accurate comparisons.

GLP-, GCLP-, and ISO-compliant biodistribution analysis

Compliance with GLP for biodistribution studies submitted to regulatory agencies ensures reliability, integrity, and traceability. As a GLP-accredited laboratory, we follow a rigorous framework to manage samples, control the work environment—including instrument maintenance, temperature regulation, staff training, and IT security—and ensure time-stamped sample handling to maintain data integrity.

GCLP bridges GLP principles with Good Clinical Practice (GCP), ensuring robust bioanalytical data in clinical settings. Meanwhile, our ISO/IEC 17025 accreditation demonstrates technical competence in laboratory testing, ensuring consistent, high-quality results. In biodistribution studies, ISO compliance further enhances credibility, particularly for non-regulated studies and global collaborations.