Accelerating the Development of Cell and Gene Therapies: How Specific Expertise and Infrastructure is Crucial in Clinical Trials
9 May 2023
As cell and gene therapy research begins to rapidly mature, with basic science discoveries making their transition into novel therapeutic products, the need for clinical trials to better adapt to the requirements of these complex studies has become evident and urgent. From manufacturing to regulatory submissions, cell and gene therapies are dictating the direction of translation and commercialisation. For the clinical trials sector, the faster the sector grows with this promising therapeutic modality, the faster life-saving and life-changing treatments will be available for patients around the world.
Cell and gene therapies mark a departure from “traditional” modalities, such as peptides, small molecules and other biologics. With both involving the delivery of highly specific biological material into patients, they directly target the underlying causes of the disease at the genetic or cellular level, rather than simply treating the symptoms. Importantly, cell and gene therapies have the potential to provide long-lasting or even curative effects in a number of different therapeutic areas, as opposed to other treatments that require ongoing use or management. Realising the promise of CRISPR-Cas9 gene editing technology and Chimeric antigen receptor (CAR) T cells, for example, will further revolutionise how we treat diseases and conditions, including cancers and rare genetic diseases.
However, cell and gene therapies are still relatively new and complex treatment modalities that require specialised expertise and infrastructure for their development, manufacture, and administration. They also present unique challenges related to safety, efficacy, and regulatory oversight, which must be carefully addressed to ensure their successful use as therapeutic agents. For the clinical trials sector, success requires a multidisciplinary team with specialised expertise in various areas, including regulatory affairs, clinical trial design, site selection, patient selection, manufacturing, clinical operations, data management and bioanalysis.
For bioanalysis of samples from cell and gene therapy clinical trials, these modalities require specialized assays to assess safety, efficacy, and mechanism of action. These should be carefully selected to address the specific questions being asked in the trial and may vary depending on the type of therapy being tested and the disease being treated. Assays can include:
- Vector quantification: Gene therapies often use viral or non-viral vectors to deliver the therapeutic gene to the patient. Assays for vector quantification can help to determine the number of vector particles or viral genomes in the product and in the patient’s body, and can be used to assess the dose and distribution of the therapy.
- Transgene expression: Assays for transgene quantitation can be used to measure the level and duration of expression of the therapeutic gene in the patient’s cells, and to assess the impact of the therapy on the target cells or tissues.
- Immunogenicity: Cell and gene therapies can trigger humoral, cellular and innate immune responses, which can impact the safety and efficacy of the therapy. Assays are often required to assess the patient’s immune response to both the vector and transgene product, as well as monitoring the development of neutralising antibodies or other immune responses that may impact the efficacy of the therapy.
- Tumour marker analysis: Cell and gene therapies may be used to treat cancer, and assays for tumour marker analysis can be used to assess the impact of the therapy on tumour growth and progression.
- Pharmacokinetics: Molecular (PCR) and flow cytometry assays are used to monitor the cell therapy in the patient’s blood through the phases of distribution, expansion, contraction, and persistence.
- Safety assays: Assays for safety can be used to assess the impact of the therapy on the patient’s cells, tissues, and organs, and to monitor for adverse effects or toxicities associated with the therapy, which requires more clinical monitoring.
- Pharmacodynamic assays: Pharmacodynamic assays that measure the mechanism of action can be used to assess the impact of the therapy on the patient’s cells and tissues, and to gain insights into the underlying biological mechanisms that contribute to the therapeutic effect.
In summary, cell and gene therapy clinical trials require specific expertise and considerations beyond those of traditional drug trials. As leaders in the field, 360biolabs understands the unique and specific needs of cell and gene therapy clinical trials. We have the protocols, infrastructure and expertise in place across our labs in Australia, North America and Europe to ensure the generation of high-quality data to support the development of these innovative and potentially life-changing therapies.