Gene Therapy and RNA

Gene Therapy and RNA

BioModels has extensive experience with a variety of gene therapy and mRNA vaccine approaches, utilizing both viral and non-viral vectors as well as ex vivo gene modified cell therapies, including AAV, plasmids, CRISPR, mRNA, RNAi, siRNA, LNPs, LNCs, and CAR-T.

We offer a fully equipped BSL-1/BSL-2 facility and customizable husbandry options to support experiments across diverse therapeutic areas, with multiple administration routes available.

Additionally, compliance with relevant national, state, and municipal regulations is ensured by the BioModels Institutional Biosafety Committee (IBC).

• Assess biodistribution and persistence of your materials via imaging (IVIS), flow cytometry, PCR, or immunohistochemistry (IHC).
• Evaluate your experimental product for efficacy and mechanism of action in a variety of translational animal models of human disease.
• Work with the BioModels team to customize or develop models most suitable for your needs.

Biodistribution

Evaluate biodistribution and persistence of your gene therapy or RNA vaccine product with BioModels’ robust imaging and endpoint analysis options, including:

• 2D imaging-based detection of bioluminescent or fluorescent compounds (IVIS® Lumina III)
• Nucleic acid extraction and PCR
• Flow Cytometry
• Immunohistochemistry (IHC)
• Custom Assays and Endpoints

Representative DataClick Image to Enlarge

Bioluminescence Imaging

Following injection of a bioluminescent substrate, animals are anesthetized and placed in the Caliper IVIS Lumina Series III (Perkin Elmer) to detect whole body bioluminescence.

Disease Models

Assess your gene therapy or RNA vaccine products for efficacy and mechanism of action in BioModels’ established diseased models. Modification or manipulation of gene expression (gene therapy), as well as introduction of RNA to direct cell production of immunogenetic proteins (RNA vaccines), are exciting new modalities for the treatment, cure, or prevention, of a number of diseases including cancer, diabetes, autoimmune/inflammatory disease, and more.

BioModels has extensive experience with a variety of preclinical modeling approaches for these diseases, utilizing standard and complex administration routes.