ATMP Analytical Development Services & CMC Support

Advanced therapy medicinal product (ATMP) analytical services & chemistry, manufacturing, and control (CMC) support including characterisation, stability and release testing

Advanced therapy medicinal product (ATMP) characterisation and analysis play an important role in providing critical chemistry, manufacturing, and control (CMC) information to be submitted in Investigational New Drug Applications (INDs). Testing is also required as part of stability studies and to support release. ATMPs are complex medicines for human use that are based on genes, tissues or cells. In addition, these may also be incorporated into a delivery technology or a medical device, for example, cells embedded in a biodegradable matrix or scaffold and are referred to as combined ATMPs.

The wide variety of ATMPs and their inherent complexity means that each will present different analytical development challenges, and so specific characterisation, potency, purity and identity assays are required for each product. Regulatory requirements for these classes of medicines are evolving in the EU and US currently and further guidance documents are expected in the near future. From a best practice perspective, there is an expectation for orthogonal analytical techniques to be applied to characterisation, stability and release testing, with methods adapted to deal with, typically, small sample volumes.

Expert ATMP Analytical Support

Our GLP / GCP / GMP laboratories have supported developers and manufacturers for over 20 years through the provision of advanced characterisation programs. We have worked on multiple studies involving cell or gene therapies, mRNA and plasmid DNA-based products. With specialist laboratory facilities, we can handle Class I and Class II Biological Agents. The facility has been designed to handle recombinant genomic materials for the purposes of research and compliant testing.

Cell and virus characterisation
Viral vector identity
Virology assays
Host cell and residual plasmid DNA
Cell-based assays/potency testing
General compendial testing
GMP analysis
GCP/GLP bioanalysis
ICH stability storage and testing
Method development and validation
QC release testing
Advanced delivery technology analytical support
Liposome drug product characterisation

Advanced analytical tools are required to overcome the challenges of characterisation, quality control, and the need to meet increasingly demanding regulatory expectations. To help our clients meet the demands of ATMP analytical development, we have adopted powerful, advanced analytical tools:

Analytical ultracentrifugation (AUC) and cryo-TEM to determine full or empty capsids
Next-generation sequencing (NGS) to confirm on-target/off-target effects, or to check whether a viral sequence has been correctly integrated into a cell line
Sequencing through difficult inverted terminal repeat (ITR) regions of adeno-associated virus (AAV) can also be achieved
Using droplet digital PCR (ddPCR) for residual DNA or viral titres
Protein purity analysis of AAV capsids by CE-SDS, with sensitive Laser Induced Fluorescence derivatization

Viral Vector Characterisation

Our experts have worked on multiple viral vector characterisation studies involving Adenovirus, Adeno-Associated Virus (AAV) and Lentivirus based products. With a wide range of expertise and technology in-house, we deliver comprehensive characterisation packages or specific services including aggregation analysis, empty vs full capsid analysis, charge heterogeneity, transgene expression, liquid chromatography and mass spectrometry (LC-MS) studies on whole virus species to characterise the viral proteome, separation of empty capsids from intact viral particles by anion exchange chromatography and digestion of isolated proteins followed by mass spectrometry to give detailed information to assist identification of viral proteins.

Supporting your Product Development

Intertek can support your product development from early-stage, through to in-process control and product release assays. Our experts are adept at developing, optimising, qualifying and validating methods for each particular class of ATMP. We also have significant experience in method transfer. With a heritage of supporting advanced pharmaceutical product development, coupled with a comprehensive range of analytical technology, our experts offer Total Quality Assurance expertise to help you ensure the safety, efficacy and quality of your ATMP product.