Cell and Gene Therapy Services

Optimized methods for cell and gene therapy products

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Cell and Gene Therapy Services

Gene Therapy Services at Frontage

Gene Therapy is a rapidly growing frontier for biological and clinical research. For products to qualify for IND (Investigational New Drug) filing, BLA (Biologics License Application) or PMA (Pre-market approval application) filing, and clinical trials, analytical testing is required to ensure that all the presented parameters and descriptors are valid. Frontage Laboratories has expanded its Analytical Biologics Group to offer Cell and Gene Therapy Services to meet all kinds of testing needs of clients. For the past 5 years, Frontage has provided analytical development, methods qualification and validation, and sample testing for our clients to help push their products forward and to help our clients succeed.

Cell and Gene Therapy

Human cell therapy is a therapy in which viable cells are injected, grafted, or implanted into a patient in order to effectuate a medicinal effect, for example, by transplanting T-cells capable of fighting cancer cells via cell-mediated immunity during immunotherapy or grafting stem cells to regenerate diseased tissues. The cells used for cell therapy are usually stem cells, which include human embryonic stem cells, neural stem cells (NSCs), mesenchymal stem cells (MSCs), hematopoietic stem cells (HSC), etc. Some differentiated cells, such as tumor infiltrated Lymphocytes (TILs) and dendritic cells (DC) are also applied well and sound promising for cell therapy.

Human gene therapy seeks to modify or manipulate the expression of a gene or alter the biological properties of living cells for therapeutic use. Gene therapy is a technique that modifies a person’s genes to treat or cure diseases. There are a variety of types of gene therapy products, including Plasmid DNA, Viral vectors, Bacterial vectors, Human gene editing technology, and Patient-derived cellular gene therapy products. The most used viral vectors for gene therapy are AAV vectors and Lentiviral vectors.

Request a quote for analytical development needs of your cell or gene therapy products.

Cell Therapy Services

Cell types for Cell Therapy

  • Chimeric Antigen-Receptor T Lymphocytes (CART)
  • Human Embryonic Stem Cells (ESC)
  • Neural Stem Cells (NSCs)
  • Mesenchymal Stem Cells (MSCs)
  • Hematopoietic Stem Cells (HSC)
  • Induced Pluripotent Stem Cell (iPSC)
  • Tumor Infiltrated Lymphocytes (TILs)
  • Dendritic Cells (DC)
  • Natural Killer Cells (NK)

Analytical Tests for Cell Therapy

Tell us about your cell and gene therapy products.

Gene Therapy Services

General Attributes and Compendial Testing

  • Appearance
  • pH
  • Conductivity
  • Osmolality
  • Volume in container
  • Particulate analysis (visible, Subvisible, HIAC)

Activity / Potency Assays

  • Physical Viral Titer by Real-time PCR (qPCR), digital PCR (dPCR)
  • Optical density (A260/280)
  • NanoSight
  • Functional viral titer by Plaque-forming assay, Fluorescence foci assay, TCID50 (end point dilution assay), Infectivity assay
  • Cell-based potency assay
  • Cytometry (Flow, FACS sorting, etc.)
  • Transduced cell viability
  • ELISA (i.e. AAV serotype, protein expression, etc.)
  • Specific Activity and Enzyme Kinetics (96 and 384 well-plate) fluorescent and absorbance assays.
  • HPLC-UV, and Ion-pairing assay
  • Gel shift assay (i.e. polymerase assay with imaging system)
  • Broken beacon (qPCR tandem plate reader polymerase assay)

Identity, Purity, and Heterogeneity

  • AAV capsid serotype by ELISA
  • Confirm the presence of viral proteome by SDS-PAGE, Mass spectrometry (MS), Western blot (immunoblot), and identity of viral vector by Genome sequencing (NGS), qPCR, dPCR
  • Purity (CE-SDS, cIEF, RP-HPLC, SEC-UV, SDS-PAGE, UV-VIS, UPLC-QTOF, UPLC-UV/FLD, CESI)
  • Marker gene expression, western blot, endpoint PCR, fluorescent, luminescent, and absorbance imaging
  • Protein/peptide mapping

Impurities

  • Host cell Related impurities / Biologics impurities by Host cell DNA / RNA / protein.
  • PicoGreen, DNA Threshold assay, qPCR, Benzonase and BSA by ELISA, TEM, Gentamicin, FBS, Trypsin, TrypLE, Glycerol
  • Capsid content (empty: full capsids) by cIEF and CESI
  • Elemental impurities (Residual plasmid by qPCR, etc.)
  • Ion analysis (Residual Cesium, etc.)
  • Process-related Impurities (QTOF; LC-MS; LC-UV; GC-FID/MSD, chromatography) (residual solvents and volatiles)

Safety

Stability

  • Visual Inspection
  • pH by Potentiometry
  • Osmolality by Osmometry
  • Aggregate formation by Light microscopy, DLS, SEC-MALS, TEM, AUC
  • Cell-based potency assays
  • Titer by q-PCR or d-PCR
  • Sterility (EP2.6.1, USP71)

Capabilities and Equipment

  • QuantStudio 5 
  • SpectraMax Plate Reader 
  • cIEF/CE-SDS 
  • LC-MS 
  • HPLC/UPLC 
  • Incubators 
  • Biological Safety Cabinets (BSL 2) 
  • Spectrophotometer 
  • AKTA (not set up) 
  • Western Blot Equipment 
  • SDS-PAGE Equipment 
  • Gel Imaging Equipment 
  • Qubit 4 Fluorometer 
  • NanoDrop Lite 
  • Microscope w/ Camera 
  • Cell Counter 
  • Walk-in Stability Chambers 
  • Reach in Stability Chambers 
  • Photostability Chamber 
  • Dissolution Apparatus

The Frontage Advantage

The CMC Biologics Team at Frontage Laboratories is composed of extremely skilled scientists who focus on quality, collaboration, and service.  Our team of expert scientists will work with you to develop and optimize analytical testing methods for cell and gene therapy methods, products, and other elements of this growing field. Frontage Laboratories can provide help with all your method development, verification, and validation needs for cell and gene therapy testing.

Get in touch with our analytical development team for your cell or gene therapy products.

Resources to Consider