Gene therapy formulations may activate the host's immune system to trigger an immune response when used, affecting the effectiveness of the drug or leading to serious toxicity problems. Therefore, immunotoxicity testing is an indispensable part of the development of gene therapy formulations, and the safety of gene therapy formulations can be better ensured by evaluating whether or not they will cause adverse immune reactions. CD Formulation provides accurate and reliable immunotoxicity testing services for gene therapies based on its advanced platform and experienced team to further ensure drug safety and regulatory compliance.

The Importance of Gene Therapy Formulation Immunotoxicity Testing

During the development process, it is critical to understand the immunotoxicity of gene therapy formulations. These viral vectors may trigger the production of antibodies in the host and trigger a cellular-level response that can have a significant impact on the final efficacy and experience of use, as well as a range of side effects. Therefore, by conducting such tests, not only can potential risks be identified, but also safety hazards can be minimized and overall stability and efficiency can be improved.

Explore Our Gene Therapy Formulation Immunotoxicity Testing

Antibody generation assessment

It is critical to assess the antibody-producing response against viral vectors. We can detect antibodies specific to viral vectors through enzyme-linked immunosorbent assay (ELISA), flow cytometry, and a number of other methods, which are used to help researchers better aid the intensity and duration of antibody immune responses within the host.

Assessment of cellular immune response

The cellular immune response is a common and focused part of the gene therapy process. The activity of T cells and natural killer (NK) cells, as well as their response to therapeutic vectors or transfected cells, can be assayed by flow cytometry, cytokine release assays, and other methods.

Cytokine storm risk assessment

Gene therapy formulations may trigger a cytokine storm during application, resulting in a severe inflammatory response in the host. The cytokine release assessment assay we provide, which integrates a variety of test conditions, can help researchers effectively and reliably assess cytokine levels, thereby predicting the risk of cytokine storms occurring, ensuring the safety of gene therapy formulations in subsequent applications, and reducing the risk of application.

Assessment of unintended immune effects

Due to individual differences and to reduce the risk of unexpected immune reactions in the application of gene therapy formulations, serologic or other humoral markers need to be tested to identify unintended immune effects and reduce the risk of adverse reactions.

Other Aspects of Gene Therapy Formulation Immunotoxicity Testing

• Laboratory animal model selection. The generally accepted experimental design for the assessment of immunotoxicity induced by gene therapy agents is to conduct serial dosing studies in rodents. Animals of both sexes should normally be used, and there should be a rationale for the use of single-sex animals. If adverse effects on the immune system are identified in routine toxicity studies, the animal species, strain, dose, duration of administration, and route of administration in additional immunotoxicity studies should be as consistent as possible with routine toxicity studies.
• Dosing design. The high dose should be higher than the dose at which no adverse effects were seen, but lower than the dose that caused the stress reaction. It is recommended that multiple doses be designed to determine the dose-response relationship and the no observed immunotoxicity dose.
• Route of administration selection. In immunotoxicity studies, the route of administration should be as consistent as possible with routine toxicity studies.

Our Technologies for Gene Therapy Formulation Immunotoxicity Testing

Highlights of Our Gene Therapy Formulation Immunotoxicity Testing

• Advanced technology platform. We have established a complete and advanced technology platform and are equipped with including flow cytometer, gene sequencer and bioinformatics analysis platform.
• Strict quality control. We have strict control standards for the entire testing process to ensure the data's authenticity, integrity, and traceability.
• Personalized service. We can provide customized solutions for immunotoxicity testing in the development of gene therapy formulations according to the specific needs of our clients.
• Rich project experience. So far, we have completed immunotoxicity tests in several gene therapy formulation development projects, and have rich experience in project management and execution.

Published Data

Technology: Immunotoxicity testing

Journal: Mol Ther

IF: 4.3

Published: 2023

Adeno-associated virus (AAV) high-dose systemic gene therapy has shown promising applications in the treatment of various genetic diseases. Despite the good results of adeno-associated virus (AAV) in spinal muscular atrophy and other diseases, there is a possibility that it may lead to death due to hepatic, renal, cardiac, or pulmonary failure in subjects. This is primarily a function of immunotoxicity due to gene therapy viral vectors. The authors of this article describe the potential mechanisms of mortality from AAV gene therapy and develop effective strategies to prevent and treat immunotoxicity induced by high-dose systemic AAV gene therapy.

CD Formulation is committed to providing high-quality and efficient immunotoxicity testing services to our clients, facilitating the safe development and application of gene therapy formulations. If you are interested in us, please feel free to contact us.