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Gene Therapy Medicinal Products

«Gene Therapy Medicinal Product (GTMP) means a biological medicinal product which fulfils the following two characteristics:

It contains an active substance which contains or consists of a recombinant nucleic acid used in or administered to human beings with a view to regulating, repairing, replacing, adding or deleting a genetic sequence;

Its therapeutic, prophylactic or diagnostic effect relates directly to the recombinant nucleic acid sequence it contains, or to the product of genetic expression of this sequence».*

Thanks to over 30 years’ experience in analytical sciences and all our laboratories (Bioanalysis (PK/TK/Immuno), Bioassays, Biochemistry, Chromatography, Elemental Impurities, Mass Spectrometry, Microbiology, Molecular Biology, Protein Characterisation) on one site, Quality Assistance covers a wide range of analytical technologies needed to support your drug development process including product characterisation, development and validation of analytical methods, stability studies, batch testing and bioanalysis to support PK/TK, biomarker and immunogenicity studies.

Our capabilities in this area are summarized in the following table :

Identity of cells by phenotypic/genotypic markers RT-PCR / Q-PCR
Presence and integrity of vector Restriction endonuclease mapping
Measurement of vector gene expression Cf. potency
General Quality
Appearance Visual inspection
Extractable volume  
Residual moisture (if lyophilised)

KF titration

Cross-contamination with other products Restriction digest / Gel electrophoresis
Transgene expression Immunoassays / SDS-PAGE / RT-PCR
Transcription unit DNA confirmation Restriction enzyme mapping / PCR
Complex identity (proteins, lipids, polymers) Peptide Mapping (UPLC (UV, QTOF, IonTrap), MALDI-TOF) / GC (MS) / IEX / RID
Plasmid, DNA identity Restriction enzyme mapping / PCR
DNA homogeneity Electrophoresis (agarose) / Bioanalyzer / CE
Genomic structural integrity PCR
Plasmid-selection markers PCR / To be discussed
Purity & Integrity
Plasmid DNA conformation (monomeric supercoiled, relaxed monomer, linear, ccc [circular covalently closed] vs oc [open circular] ) Electrophoresis (agarose) / CE
Optical density ratio (A260/A280) Spectrophotometer
Viral protein profile (defective or immature particles) (U)HPLC (UV, MS)
Structural protein analysis (U)HPLC (UV, MS) / SDS-PAGE / Peptide Mapping (UPLC (UV, QTOF, IonTrap), MALDI-TOF)
Complex purity (proteins, lipids, polymers) Peptide Mapping (UPLC (UV, QTOF, IonTrap), MALDI-TOF) / GC (MS) / IEX
(Viral-) vector purity (U)HPLC (UV) / Spectrophotometer
Viral capsid purity SDS-PAGE
Homogeneity of size Electrophoresis (agarose)
Total protein Micro BCA
Gene-vector titer, infectious titer Bioassays / Q-PCR
Plasmid DNA weight, DNA titer UV / PicoGreen / Q-PCR
Viral DNA copies Q-PCR
Vector copy number ELISA / Q-PCR
Labeled dose UV / Q-PCR
Function of expressed gene Cell-based assays
Infection, transfection/transduction Cell-based assays / Q-PCR
Transgene expression after transfection in cells Cell-based assays / Western Blot / ELISA / RT-PCR
In Vitro viral genome copy number in cells Q-PCR
Product-related Impurities
Vector aggregates A4F/HF5 (UV, RI, MALS) / DLS
Transgene product Cf. Product sheet «Proteins»
Process-related Impurities
Residual DNA (bacterial chromosomal DNA, genomic DNA, plasmid DNA, HCDNA) Q-PCR
Residual proteins (HCP, bacterial proteins) (SDS-)PAGE / Western Blot / ELISA / ECL (MSD)
Medium components (FBS, antibiotics, cytokines, hormones, BSA, enzymes, antibodies, growth factors) ELISA / ECL (MSD) / (U)HPLC (UV, FLUO, MS) / Luminex
Process contaminants (serum, CsCl, EtBr, Iodixanol, alakaline detergents, acetate, PEG) CE / GC (MS) / (U)HPLC (MS) / ELISA / ICP (MS)
Residual benzonase ELISA
Residual solvents HS-GC (FID, MS) / (U)HPLC (UV)
Residual salts (U)HPLC (conductimetry) / IEX (conductimetry) / ICP (OES, MS)
Elemental impurities ICP (OES, MS)
Microbiology / Endotoxins Bioburden / Sterility (filtration, direct inoculation) / Endotoxins (kinetic, end point)
Container closure system integrity Bubbling / Dye ingress  / Microbial ingress
Leachables GC (FID, ECD, MS) / ICP (OES, MS) / (U)HPLC (UV, ELSD, CAD, MS)
Cytotoxicity / Biological reactivity Cell-based assays / USP <87>
Immunogenicity & PK/TK & Biomakers
Immunogenicity (on transgene product) ELISA / ECL (MSD) / Cell-based assays (neutralization) / Luminex / Biacore
PK/TK / Biodistribution / Shedding Q-PCR / ELISA / ECL (MSD) / Luminex / Biacore
Biomarkers ELISA / ECL (MSD) / UPLC (MS/MS) / Luminex
Vehicles / Buffers Q-PCR / UV

* Reflexion paper on classification of advanced therapy medicinal products, 7 December 2012, EMA/CAT/600280/2010

You can download the full table of GTMP analysis here!

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