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Gene Therapy Assays Customized Gene Therapy Assays/Tests

From idea to IND/NDA/BLA – Customized services and license-free assay development for gene therapy development.

Gene therapy corrects or modifies and existing gene or introduces a new gene to treat or prevent diseases. There are two basic types of gene therapy, somatic gene therapy and germ-line gene therapy. In somatic gene therapy, cells are modified where the corrected or modified gene is non-inheritable. In germ-line gene therapy DNA or RNA are introduced into germ cells where the modified gene is inheritable (passed onto next generations. This therapy is still controversial and typically requires ethics committee review.

Fig. 1 Gene therapy methods


The two types of gene delivery methods are: 1) in vivo transfer of a gene to cells inside a body and, 2) ex vivo gene transfer where patient cells are isolated, gene is transferred in vitro, and these modified cells are transferred back into the patient.

Marin Biologic Laboratories (“Marin Bio”) offers customizable R&D, pre-clinical and clinical assay services for gene therapy development. Our customized services also include development of project-based innovative solutions or technologies for our clients. Our clients own the technologies we developed for them (certain restrictions may apply).

Gene therapy pre-clinical, clinical and GMP assays we offer:

Molecular biology & qPCR

  • Viral vectors & plasmids construction (research grade & small-scale GMP grade)
  • Infectious genome and vector genome titers
  • Residual host DNA quantitation

ELISA & Immunoassays

  • Protein titer by ELISA
  • Residual host protein quantitation
  • Pharmacokinetics (PK) assays
  • Pharmacodynamics (PD) assays

Cell-based assays for gene therapy

  • Gene-specific functional assays
  • Lymphocyte activation assay
  • Transduction inhibition (TI) assay
  • Reporter assays
  • Development of customized reporter assays

Immunogenicity assays

  • ADCC, Antibody-Dependent Cellular Cytotoxicity bioassays
  • ADA, Anti-Drug Antibody inhibiting drug action
  • T-cell assays: Peripheral blood mononuclear cell (PBMC)- and dendritic cell (DC)- based assays.
  • Cytokine Release Syndrome Assay
  • Natural Killer (NK) Cell Assay
  • Phagocytosis Assay

Flow cytometry assays for gene therapy

Custom stable cell line development

Master cell bank

  • Production and characterization of a master cell bank for vector production and cell-based assays.

Drug lot release assays/tests

Following assays or tests are offered for AAV vector-based drug lot release:

Assay type Technique/method used
Potency assays Cell-based assays, Flow cytometry,
Cell-based ELISA
Viral genome titer q-PCR
Viral infectivity Cell-based assays
Recombinant expression In vitro transduction/transfection
Protein purity SDS-PAGE or Mass spectrometry
Nucleic acid contamination Spectrophotometry
Residual host-cell DNA q-PCR
Residual plasmid DNA q-PCR
Residual host cell proteins ELISA
Residual host cell nucleic acids q-PCR

Potency assays for gene therapy drugs

Technologies used for potency assays:

Case study : Development of potency assay for adeno-associated virus (AAV) gene therapy drug

Goal: Cell-based potency assay using an adeno-associated virus (AAV) gene therapy drug which binds a killer T-cell line to a cancer cell line and measures cell killing.

Method: This experiment compares commercial Reference Standard to Test Sample (bifunctional or bispecific antibody contruct). The target cancer cells are pre-loaded with a fluorescent dye. The effector killer cell and target cancer cell lines are incubated at a specific effector cell-to-target cell concentration. Cells are incubated with increasing concentrations of bispecific antibody contruct. When target cancer cells are killed, the fluorescence released into the supernatant is measured (Fig. 2).

Fig. 2: Cell-based potency assay using an adeno-associated virus (AAV) gene therapy drug


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