Cell and Gene Therapy Development

Cell and Gene Therapy Development


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.


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Issues with Approval

The issues companies face when manufacturing these type of biological produces is that they are introducing a variability right away in the process in terms of the active ingredient. In type of manufacturing, the active ingredient is the specifics patient’s genome. With the variable ingredient, manufactures do not know what they are working with up front 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 companies without experience in this area.


The Non-traditional Approach

This approach to the drugs approval is based on individualized patients genetic makeup and requires the developers to make a constant process of taking the patients genetic material and produce the proper individualized 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 event though it is based on their own genetic material.


About RCA®

Regulatory Compliance Associates® (RCA) provides healthcare consulting services to the following industries for resolution of compliance and regulatory challenges:



We understand the complexities of running a life science business and possess areas of expertise that include every facet of R&D, operations, regulatory affairs, quality, and manufacturing. We are used to working on the front lines and thriving in the scrutiny of FDA, Health Canada, MHRA and globally-regulated companies.


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About Sotera Health

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Sotera Health Company, along with its three best-in-class businesses – Sterigenics®Nordion® and Nelson Labs®, is a leading global provider of mission-critical end-to-end sterilization solutions and lab testing and advisory services for the healthcare industry. With a combined tenure across our businesses of nearly 200 years and our industry-recognized scientific and technological expertise, we help to ensure the safety of over 190 million patients and healthcare practitioners around the world every year.


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