Cell and gene therapy (also known as “CGT”) is an emerging field that offers new opportunities for treating a wide range of diseases, including cancer, genetic disorders, and autoimmune diseases. Unlike traditional pharmaceutical drugs, cell and gene therapies involve using living cells or genetic material engineered to treat specific diseases. As such, CGT’s complexity requires a comprehensive analytical approach to ensure the therapies’ safety, purity, and efficacy.
Analytics plays a pivotal role in cell and gene therapies, with multiple categories arising within the development process. Categories include disease history and progression, biomarker identification and validation, identity and characterization, among others. Each category represents unique challenges and requires a tailored, rigorous approach.
Despite the scientific rigor built into the research process, there remains an element of subjectivity within this still-experimental process. CGT researchers typically rely on a decision-tree approach that makes it difficult to identify a single set of consistent assays or tests to use. Often, researchers ask conditional questions, such as “If we modify this gene, will it result in the desired therapeutic effect?”
A new field also introduces a novel set of risks. Among the chief concerns of cell and gene therapy is the potential for adverse immune reactions. CGT therapies involve the use of living cells or genetic material. Therefore, there is a risk that the body’s immune system will recognize them as foreign and mount a response. This can result in serious side effects and even death.
Cell and gene therapies, by definition, present a constant risk for contamination. Given CGT’s use of living cells, the potential for bacterial or viral contamination is constant, compromising the efficacy of the therapy, and even introducing a life-threatening risk.
To address these and other concerns, CGT analytics must be comprehensive, robust, and tailored to the specific therapy being developed. This requires a deep understanding of the underlying biology and biochemistry of the therapy. Additionally, researchers should have a mastery of a wide range of analytical techniques, including genomics, proteomics, and flow cytometry.
However, given the newness of cell and gene therapies, researchers are often stymied by a lack of standardization. There are no set guidelines or best practices for CGT analytics. The FDA is working with industry leaders to develop guidance documents outlining the analytical testing and questions that should be addressed throughout the CGT application workflows.
The few cell and gene therapies currently on the market have undergone years of development and rigorous regulatory hurdles, and serve as templates for future work.
While there is still much to learn about the field, regulatory-approved CG therapies provide a framework for best practices and standards that will guide future research and development. With continued innovation and investment, CGT has the potential to revolutionize medical care and improve the lives of millions.
Cell and Gene Therapy Balances Promise and Risk
