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.
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).
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,
|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|
Technologies used for potency assays:
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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“Parts of the figure were drawn by using pictures from Servier Medical Art. Servier Medical Art by Servier is licensed under a Creative Commons Attribution 3.0 Unported License (https://creativecommons.org/licenses/by/3.0/).”