Services → Pharmacodynamics

Pharmacodynamic biomarker analysis in cell and gene therapy

GLP-accredited and GCLP-compliant pharmacodynamic analyses to assess drug effects, including PD biomarker quantification, drug target engagement, vector functionality, transgene expression, CAR-T expansion, and immune activation.

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PD BIOMARKER ASSAY DEVELOPMENT

Pharmacodynamic analysis services:

Transgene expression

Custom, validated qPCR and dPCR assays—from nucleic acid extraction and cDNA conversion in the RT step to high-throughput analysis—designed to detect transgene expression in vivo in modified cells, AAV transgene expression, and other gene expression profiles. GLP, GCLP or non-GLP compliant services.

RNA expression profiling for pharmacodynamic biomarkers

High-quality RNA extraction and RNA-Seq technologies identify upregulated and downregulated genes as biomarkers for drug response, pharmacodynamic transcript biomarkers, efficacy evaluation, pathway activation, and other pharmacodynamic assessments.

Immune activation

Monitor immune activation across multiple stages by quantifying single-gene expression using qPCR/dPCR, profiling T-cell and B-cell clonality from RNA or DNA, and measuring cytokine and inflammatory protein levels from just microliters of blood or plasma.

Pharmacodynamic analysis of cell expansion in cell therapy

Using custom qPCR/dPCR assays, we detect and quantify cell therapy persistence and in vivo expansion—including T-cell, B-cell, stem cell and NK-cell expansion—for pharmacodynamic assessment in blood and tissues. GLP, GCLP or non-GLP compliant services.

Pharmacodynamic protein biomarkers

Using affinity-based proteomics, we quantify proteins across a wide range of expression levels in a highly multiplexed manner in blood and plasma to detect drug response, target engagement, and other pharmacodynamic changes. Available as focused plasma protein assay panels with fewer targets for high statistical power or as larger exploratory panels for maximized discovery.

A comprehensive guide

PK/PD assays in gene therapies

PK/PD assays in gene therapies are crucial for understanding inter-patient variability, persistence, and long-term dynamic effects post-administration. Robust and efficient assays are essential for this. Download the guide to learn more.

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PK/PD assay development

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

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GLP and GCLP 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. In addition, we are Good Clinical Laboratory Practice (GCLP) compliant to ensure the safe and reliable analysis of clinical samples.

Pharmacodynamics at TATAA

Samples

Blood
Tissues
Biofluids

Test items

Total RNA
mRNA, miRNA
Genetically engineered cells
Plasma proteins categorized by pathways and disease areas
Inflammatory proteins, including cytokines and chemokines

Pharmacodynamic analysis in cell and gene therapy

For transgene expression and vector functionality, validated custom qPCR or dPCR assays detect transgene transcripts while distinguishing them from endogenous expression or vector DNA. These assays are designed to quantify vector copy numbers, enabling the monitoring of vector persistence and biodistribution.

We assess cytokine and immune activation protein biomarkers using affinity-based high-plex panels on plasma or blood, applicable to both clinical and preclinical samples.

RNA-Seq monitors gene expression changes and functional signatures at the RNA level relative to baseline to understand pathway activation.

Pharmacodynamic assays in cell and gene therapy

The choice of pharmacodynamic assay depends on the required sensitivity. qPCR and dPCR enable highly sensitive detection of transgene-expressed RNA down to just a few copies in the most sensitive assays. However, transgene proteins may be challenging to detect at such low copy numbers, making mRNA a useful proxy for protein expression.

If downstream pathway activation is to be monitored, RNA-Seq assesses transcript-level changes, while affinity-based protein panels provide insights into protein-level changes.

TCR/BCR sequencing is used to monitor clonal expansion in CAR-T therapies.

Immune monitoring can be performed using a limited set of biomarkers with qPCR/dPCR, offering high throughput and focused data, or through comprehensive transcript profiling using RNA-Seq. Cytokines and immune proteins can be quantified with absolute quantification using targeted affinity-based proteomics panels, requiring only microliters of human plasma or blood.

Pharmacodynamic biomarkers in cell and gene therapy

Pharmacodynamic (PD) biomarkers in cell and gene therapy are critical for assessing the mechanism of action, therapeutic response, durability, and safety.

PD biomarkers for gene and transgene expression include transgene mRNA levels in AAV, lentiviral (LV), and mRNA-based therapies. Additionally, changes in endogenous gene expression due to treatment and immune response-related gene expression, such as cytokines or immune checkpoint markers, serve as key PD biomarkers.

In cell therapies, PD biomarkers include cellular persistence and expansion, measured by CAR transgene copy numbers in T cells, TCR/BCR clonal profiling to track dominant clones post-therapy, and vector copy number (VCN) in gene-modified cells.

Cytokine and chemokine profiling involves monitoring inflammatory and immunosuppressive markers using qPCR, dPCR, or NGS. Additionally, affinity-based proteomics allows the quantification of cytokines and other inflammatory proteins from microliters of blood or plasma, providing crucial insights into immune responses.

GLP, GCLP, and ISO-compliant pharmacodynamic analysis

Data reliability, integrity, and traceability are critical for pharmacodynamic (PD) biomarker analysis in cell and gene therapy development. As a GLP-accredited laboratory, we adhere to stringent regulatory standards for sample management, controlled work environments, instrument maintenance, temperature regulation, staff training, and IT security. Our workflows incorporate time-stamped sample handling, preserving sample and data integrity throughout analysis.

Good Clinical Laboratory Practice (GCLP) integrates GLP principles with Good Clinical Practice (GCP), ensuring high-quality, regulatory-compliant bioanalytical data for clinical PD biomarker studies. This alignment is essential for tracking treatment responses and validating therapeutic efficacy in clinical trials.

Our ISO/IEC 17025 accreditation reflects our technical competence in laboratory testing, ensuring reproducible, high-quality results. In pharmacodynamic biomarker analysis, ISO compliance reinforces credibility, particularly for non-regulated studies and global collaborations, supporting the advancement of gene and cell-based therapies through robust and reliable bioanalytical insights.