Cell and Gene Therapies show promise to enhance treatment of diseases

by Katherine Bergmann, Ph.D., Manager of Viral Safety & Viral Clearance

The development of Advanced Therapy Medicinal Products (ATMPs), such as gene and cell therapy products, has made significant progress in the treatment of many diseases, including cancer, genetic, and autoimmune disorders. Regulatory agencies have seen a large increase in the number of submissions over the last two years, and further increases are expected to continue for years to come. With the promise to enhance treatment, greatly reduce side effects, and potentially cure many types of diseases and disorders, these therapies are in high demand, and biopharma companies are in a race to the clinic.

However, these technologies are very complex in nature and are vastly different than traditional biopharmaceutical products, especially when it comes to the use of these products for personalized medicine. The complexities span the development pipeline, creating challenges for manufacturing, testing requirements, regulatory approval, and commercialization.

In gene therapies, a defective gene is replaced with a functional one using a transmission system called a vector. This may occur by injecting the vector into the patient, or cells may be removed from the patient, exposed to the vector, and then returned to the patient.

In some cases a single therapy may be used for many patients with the same condition. In other cases, there may be a custom treatment for each patient (known as personalized medicine).

Gene therapies provide the ability to treat genetic diseases such as hemophilia by a single gene therapy treatment, in contrast to repeated treatments needed with conventional therapies. In addition to enhanced treatments, other potential benefits include reduced side effects, and the potential to cure, rather than just treat, many types of diseases and disorders. With cell therapies, human or animal cells are administered to “repair, replace, regenerate, or augment a recipient’s cells, tissue, or organs that are diseased, dysfunctional, or injured” (USP <1046>). The cells may be used without alteration, or they may be engineered to add a specific function (for example, using CRISPR or CART technologies). Cell therapies may be used for disorders such as knee cartilage defects or organ failure, where cells can be implanted that will replace the damaged tissue.

Eurofins BioPharma Product Testing supports the development of ATMPs both for traditional use as well as for use in personalized medicine.

Eurofins provides comprehensive GMP-compliant CMC testing support to ensure the identity, potency, purity, and safety of starting materials, cell and virus banks, intermediate products, vectors, and final drug products, as well as support for manufacturing process development and validation.