What Is A Cell & Gene Therapy? 

Cell & gene therapies are a group of treatments that use cells and/or genes to heal damaged tissues and organs. The goal of cell & gene therapy is to treat genetic diseases by correcting the errors in the genome that cause them. Regulatory authorities such as the FDA regulate cell & gene therapy products and granted the market authorization of several products for patient use (1). As such, CGT was often approved for use in patients suffering from hematological malignancies (e.g., relapsed or refractory multiple myeloma), solid tumors (e.g., unresectable or metastatic melanoma), and other rare genetic disorders (1, 2). 

Did you know that Cerba Research was involved in the market authorization of 4 CGTs including 3 chimeric antigen receptor T cell therapy (CAR-T) products? Check out our wide range of integrated specialty and safety testing capabilities below and get in touch to learn more. 

Your Next-generation Cell & Gene Therapy Development Partner

The fast-paced cell & gene therapy landscape brings the potential for health improvements for patients waiting for appropriate personalized treatments. As such, cell & gene therapy biotech and pharma communities face the challenge of bringing novel therapeutics to the market within tight deadlines. Meeting the demands of cell & gene therapy development at the clinical translational stage of the drug development process requires a strategic partner capable of dealing with highly complex clinical trial designs and laboratory tests. The industry needs highly customized laboratory solutions for their next-generation cell & gene therapy developments to deal with this complexity.

We Perform Specialty Testing In ~95% Of Our Cell & Gene Therapy Portfolio

Cerba Research is uniquely positioned to support your CGT trial with a wide range of in-house laboratory solutions depending on your specific indication. Thanks to our expertise in assay development, validation, customization capabilities, robust kit building, sample management, and logistics, we have executed upon about 50 CGT trials since 2018 and growing. Cerba Research performed cell & gene therapy trials in indications such as multiple myeloma, acute myeloid leukemia, colorectal cancer and epidermolysis bullosa amongst others.

Additionally, Cerba Research can offer tailor-made solutions for your CAR-T and other cell & gene therapy and help you design and validate specialty-based assays aligned with the industry’s complex FDA guidance for the industry (4) as outlined below.

We offer customized assay design and fit-for-purpose validation. Cerba Research works with the biotech and pharma communities as a fully integrated partner, using its knowledge and scientific expertise to help sponsors shape testing protocols and identify the right testing strategy to secure successful outcomes. Upon initial validation in the EU or US region, assays can be transferred and implemented around the globe, according to the need of the trial. Cerba Research offers a full suite of immunology, genetics, genomics, and flow cytometry assay solutions as indicated below.

Central Laboratory & Clinical Trial Logistics For Cell & Gene Therapy Development

Our unique combined specialty laboratory and central lab services offer the best of both worlds, allowing our customers to have one partner for all test services. From choosing the most appropriate geographies and labs to our best-in-class logistic network, securing the integrity of samples. Cerba Research’s fully integrated specialty logistic portfolio includes the following: 

• Accessibility to a global network of laboratories capable of performing complex specialty testing for cell & gene therapies.

• Global PBMC / BMMC isolation network, securing sample viability and integrity for further downstream analysis.

• Samples are monitored 24/7, temperature-controlled, and transported to reach their destination on time. 

• Kit manufacturing, assembly, and worldwide distribution to support your CGT trial.

• Highly customized global logistics solutions for complex cell & gene therapy development challenges. 

Adeno-associated Virus 9 (AAV9) Testing

At Cerba, we are performing routine anti-AAV9 antibody testing. The anti-AAV9 human IgG ELISA-based testing is intended for an in vitro semi-quantitative estimation of anti-AAV9 IgG antibodies titers directed against AAV9 capsid particles present in the patient’s serum sample. This assay is intended to assess the presence of anti-AAV9 antibodies in patients (including newborns and infants) diagnosed with SMA. Antibody titer levels are used to make an appropriate treatment decision, as high titers prohibit administering an appropriate gene therapy.

Cerba Research is your go-to partner for tailored logistic solutions for your next-generation of cell & gene therapies. Here, you can read more about our capabilities to set up and operationalize a highly customized gene therapy targeting spinal muscular atrophy (SMA), a rare genetic disease affecting the central and peripheral nervous system and voluntary muscle movement (2).

Immunogenicity In CGT Trials

Our clients are supported from pre-clinical to post-market authorization with our in-depth biologics and biosimilar experience. We offer long-standing scientific expertise in PK and immunogenicity (antibody-drug antibodies (ADAs) and NAbs) with a standardized or customized approach as per your vision.

Our focus extends from monoclonal antibodies to cell & gene therapies and more. In addition, we are Good Laboratory Practice (GLP), the College of American Pathologists (CAP), and Clinical Laboratory Improvement Amendments (CLIA) accredited for your regulatory requirements.

References

1. U.S. Food & Drug Administration: Approved cellular and gene therapy products. URL [Approved Cellular and Gene Therapy Products | FDA].

2. Orphanet. URL [Orphanet].

3. U.S. Food & Drug Administration: Cellular & gene therapy guidances. URL [Cellular & Gene Therapy Guidances | FDA].