Cell and Gene Therapies also known as (ETMPs) have continued to evolve in recent years. Over the last 15 years the industry and regulatory bodies have continued to learn more about them and in recent year there have been more and more guidance documents released outlining what the regulating bodies want the manufacturers and developers of these different biologic products to look for and adhere to during development.
Cell and Gene Therapies are Personalized medicine meaning, one patient, one drug. These types of drugs are not produced in large scale or in batches like the standard biologics or drugs we have been used to manufacturing up to this point. They are made in very small batches that are produced in laboratory setting but require the same environmental controls as your typical aseptic processing but a much smaller scale.
Issues with Approval
The issues companies face when manufacturing cell and gene is that they are introducing variability right away in the process in terms of the active ingredient. In this type of manufacturing, the active ingredient is the specific patient’s genome. With the variable ingredient, manufacturers do not know what they are working with upfront which makes the process difficult. It is a non-tradition way of thinking that makes validating a process that has that kind of variability hard on cell and gene companies without experience in this area.
The Non-traditional Approach
This approach to drug approval is based on an individualized patient’s genetic makeup and requires the developers to make a constant process of taking the patient’s genetic material and producing the cell and gene therapy dose at the end. Developers have to be able to categorize the variable, walk it through the process, and then categorize it again at the end making sure it is safe and effective for the patient even though it is based on their cell and gene genetic material.
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